NeurologyLive Mind Moments

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, "Bemdaneprocel and the Future of Cell Therapy in Parkinson Disease" Rajesh Pahwa, MD, director of the Parkinson's Disease and Movement Disorder Center at the University of Kansas Medical Center, discusses the phase 3 exPDite-2 trial evaluating bemdaneprocel, an investigational cell therapy for Parkinson disease (PD). Pahwa explains the rationale behind cell replacement therapy as a means to restore dopamine production, outlining the progression from fetal tissue implants to stem cell–derived dopaminergic neurons. He details key elements of the study design, including patient selection criteria, trial endpoints, and long-term goals. Reflecting on decades of research in this space, he emphasizes the potential impact of a one-time, hardware-free surgical option and how this novel approach could complement or shift the current treatment landscape. While many questions remain, Pahwa highlights the promise of bemdaneprocel as a next-generation option in Parkinson care. Looking for more Movement disorder discussion? Check out the NeurologyLive® Movement disorder clinical focus page. Episode Breakdown: 1:00 – Mechanism and rationale behind bemdaneprocel as a dopaminergic cell therapy 3:00– Key design considerations for conducting a registrational phase 3 cell therapy trial 5:25 – Trial structure, eligibility criteria, and ongoing patient recruitment 6:35 – Neurology News Minute 8:45 – Significance of reaching phase 3 with a cell therapy in Parkinson disease 10:20 – Future role of cell therapy within the Parkinson disease treatment landscape 12:10 – Unique aspects of the exPDite-2 trial and what sets it apart from earlier efforts The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: BTK Inhibitor Fenebrutinib Demonstrates Long-Term Suppression of MS Activity in Open-Label Extension REGENXBIO Reports Positive Phase 1/2 Data for Higher Dose of RGX-202 in Duchenne Muscular Dystrophy NewAmsterdam Pharma Reports Positive Topline Data of Alzheimer Agent Obicetrapib from Phase 3 BROADWAY Trial Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, "World MS Day: Raising Awareness, Evolving Care, and Empowering Patients," Taylor Hess, MD, a multiple sclerosis specialist at the Michigan Institute for Neurological Disorders (MIND), shares insights in recognition of World MS Day. She reflects on the importance of raising global awareness for MS, the need for equitable care, and the evolving focus of research and treatment. Dr. Hess discusses underrecognized symptoms like fatigue and cognitive dysfunction, and emphasizes the value of the upcoming diagnostic criteria update in enabling earlier intervention. She highlights key research priorities—including progressive MS, symptom management, and remyelination—and speaks to the importance of holistic, lifestyle-informed care. Finally, she shares advice for clinicians on how to communicate a new MS diagnosis with empathy, clarity, and empowerment. Looking for more multiple sclerosis discussion? Check out the NeurologyLive® Multiple sclerosis clinical focus page. Episode Breakdown: 1:00 – Significance of World MS Day and the importance of global MS awareness 2:05– Commonly recognized vs overlooked MS symptoms in patient care 3:10 – Transitioning to updated MS diagnostic criteria and the need for global provider education 4:15 – Neurology News Minute 6:30 – Research priorities: progressive MS, symptom management, and remyelination strategies 7:35 – The role of lifestyle, risk factor control, and holistic care in MS management 8:50 – Communicating MS diagnosis with empathy, support, and resource guidance The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves First Autoinjector Form of DHE for Acute Migraine and Cluster Headache FDA Clears Lumipulse Plasma Ratio as First Blood Test for Diagnosing Alzheimer Disease Once-Daily Oral Pill for Obstructive Sleep Apnea Shows Promising Phase 3 Results, Paving Way for FDA Submission Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, "Refining TSC Care: Phenotyping, Clustering, and Clinical Impact," Ajay Gupta, MD, director of the Tuberous Sclerosis Program at Cleveland Clinic, discusses recently published research that used unsupervised clustering to group over 900 patients with tuberous sclerosis complex (TSC) into four clinically meaningful phenotypic clusters. He outlines the distinct traits of each cluster—ranging from tumor risk to cognitive impairment—and explains how variant-specific genetic data helped reinforce these categories. Gupta, who also serves as a professor of neurology at the Cleveland Clinic Lerner School of Medicine, also explores the clinical value of these findings for surveillance planning, early intervention, and future therapeutic trials. He emphasizes that while overlap between clusters exists, this approach lays essential groundwork for precision care and more targeted research in TSC. Looking for more epilpesy discussion? Check out the NeurologyLive® Epilepsy clinical focus page. Episode Breakdown: 1:00 – Study goals and the shift from genotype-to-phenotype toward phenotype-to-genotype mapping 2:40– Overview of the 4 main phenotypic clusters identified in the TSC population 8:05 – Genetic domain associations and their impact on clinical monitoring and treatment 11:50 – Neurology News Minute 14:45 – Understanding overlap between clusters and avoiding overprediction in clinical settings 17:00 – Implications for future surveillance strategies and precision candidate selection in TSC trials The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA AdComm Plans to Review Investigational Cell Therapy Deramiocel for DMD Cardiomyopathy Gene Therapy AAV-GAD Gains Regenerative Medicine Advanced Therapy Designation as Potential Parkinson Treatment Microbiome-Targeting Therapy MaaT033 Continues to Show Promise in Final Phase 1 Readout Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, "Down Syndrome and Alzheimer's: Clinical Trials, Equity, and Patient-Centered Progress," Elizabeth Head, PhD, professor in the department of Pathology and Laboratory Medicine at the University of California, Irvine, discussed the evolving intersection of Down syndrome and Alzheimer disease using updates from the Alzheimer Biomarker Consortium – Down Syndrome (ABC-DS). She detailed how outcome assessments have been refined to meet the unique cognitive profiles of individuals with Down syndrome and shared the progress in launching clinical trials that now include this historically excluded population. Head also addressed the challenges in distinguishing developmental delay from early dementia symptoms, the potential role of anti-amyloid therapies, and how non-pharmacological interventions like sleep apnea treatment may contribute to cognitive preservation. She emphasized the importance of equitable research participation, clinician education, and understanding patient priorities—like independence and respect—when supporting individuals with Down syndrome and their families. Looking for more Alzheimer disease/dementia discussion? Check out the NeurologyLive® Alzheimer disease/dementia clinical focus page. Episode Breakdown: 1:00 – Overview of the ABC-DS initiative and the growing inclusion of Down syndrome in Alzheimer research 2:00– Current state of clinical trials and progress in therapeutic development for this population 3:30 – Evolution of outcome assessments tailored for individuals with Down syndrome 5:40 – Challenges in distinguishing Down syndrome traits from signs of early dementia 7:40 – Clinician educations needs and improving access to specialized care 9:00 – Neurology News Minute 11:00 – Eligibility and considerations for anti-amyloid therapies in Down syndrome 13:35 – Research gaps, co-occurring conditions, and the importance of reaching underserved populations 16:40 – Top priorities from patients and families: independence, inclusion, and respect in care settings The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: GENERATION HD2 Trial of Huntington Agent Tominersen Amended to Include Only Higher Dose FDA Grants Authorization to Epiminder's Implantable Continuous EEG Monitor for Epilepsy Treatment FDA Approves FcRn Blocker Nipocalimab for Broad Forms of Generalized Myasthenia Gravis Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, "Sleep and Seizures: Emerging Insights From AAN 2025," Nancy Foldvary-Schaefer, DO, director of the Sleep Disorders Center at Cleveland Clinic, reflected on key sleep-related topics presented at this year's AAN Annual Meeting. She highlighted the growing clinical interest in underrecognized conditions like idiopathic hypersomnia and narcolepsy, stressing the need for better awareness and earlier diagnosis. Foldvary-Schaefer also detailed the development of the Sleep by Cleveland Clinic mobile app, designed to improve screening for common sleep disorders with validated tools and real-time resources. Additionally, she discussed new findings on the prevalence of obstructive sleep apnea in adults with epilepsy, pointing to the importance of routine sleep screening in this population, regardless of seizure severity. Finally, she shared exploratory data on SSRI use and peri-ictal respiratory patterns in epilepsy, offering new perspectives on potential protective mechanisms tied to SUDEP risk. Looking for more neuromuscular discussion? Check out the NeurologyLive® Sleep disorders clinical focus page. Episode Breakdown: 1:00 – Key sleep topics discussed at AAN 2025 and the growing interest in hypersomnia 5:45 – Development and purpose of the SLEEP mobile app by Cleveland Clinic 10:35 – Findings on obstructive sleep apnea prevalence in adults with epilepsy 15:05 – Neurology News Minute 17:10 – The need for sleep disorder screening in epilepsy clinical trials 19:45 – Exploratory findings on SSRI use and respiratory patterns in patients with epilepsy The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Pre-Filled Syringe Administration for FcRn Modulator Efgartigimod FDA Approves CT-132 as First Digital Therapeutic for Preventive Treatment of Episodic Migraine FDA Expands Diazepam Nasal Spray Indication to Treat Ages 2 to 5 Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, "Training the Next Generation of Neurology Nurse Practitioners," Daniel Harrison, MD, neurointensivist and assistant professor of neurology at Boston Medical Center, discussed his role as a senior author on a paper focused on a national needs assessment survey of neurologic education in nurse practitioner (NP) programs. In the conversation, Harrison explained how the survey aimed to understand neurology education across NP programs and identify gaps in training for advanced practice providers. He highlighted key findings, including the importance of lesion localization and neuroradiology for NP postgraduate training. While basic neurology education is integrated into many NP programs, he emphasized the need for more hands-on clinical experiences in specialized areas like neurocritical care and neuro-oncology. He also discussed the critical shortage of preceptors for neurology rotations, which hinders clinical education. Looking ahead, Harrison shared his thoughts on the growing role of NPs in neurology, forecasting a substantial increase in their numbers over the next decade and its impact on both clinical care and education. He also acknowledged the contributions of his colleague, Kate Sweeter, NP, who played a key role in the research, highlighting the importance of NPs in clinical care and academic advancements in neurology. Looking for more neuromuscular discussion? Check out the NeurologyLive® Neuromuscular clinical focus page. Episode Breakdown: 1:00 – Overview of the national needs assessment survey  3:45 – Importance of lesion localization and neuroradiology for NP postgraduate training 6:05 – The role of neuropathology in neurology education for nurse practitioners 8:05 – Challenges with clinical rotation preceptors and the shortage in neurology 9:40 – Neurology News Minute 12:10 – The role of NP programs in providing clinical experience 14:55 – Variability and lack of standardization in NP neurology education 18:05 – Evolving duties and responsibilities of NPs over the coming years 19:35 – Closing thoughts, acknowledging the contributions of Kate Sweeter, NP, and the value of NPs in neurology The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Accepts Regulatory Submission for BTK Inhibitor Tolebrutinib for Non-Relapsing Secondary Progressive MS FDA Approves Diazoxide Choline Extended-Release Tablets for Hyperphagia in Prader-Willi Syndrome Genentech Reports High-Dose Ocrelizumab Fails to Show Additional Benefit in Relapsing MS Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we cover the recent approval of Supernus Pharmaceuticals' agent SPN-830, marketed as Onapgo, as the first and only subcutaneous apomorphine infusion device for the treatment of motor fluctuations in adults with advanced Parkinson disease (PD). SPN-830, which has been available in Europe for several years, was approved based on data from the TOLEDO study (NCT02006121), a randomized, double-blind study in which treatment with the device was associated with a difference of –1.89 hours per day of OFF time for patients with PD in comparison with placebo. To gain greater insights on how this approval impacts the clinical and patient communities, NeurologyLive® sat down with movement disorder experts Stuart Isaacson, MD, director of the Parkinson's disease and movement disorders center of Boca Raton, and Rajesh Pahwa, MD, the Laverne and Joyce Rider professor of neurology at the University of Kansas Medical Center. The duo discussed the significance of the approval, considerations for patient selection with the treatment, as well as the studies supporting its safety and efficacy. In addition, the pair provided context on how this approval, along with other recent approvals in PD, speak to the progress the clinical and research community has made over the years. For more of NeurologyLive's coverage of SPN-830 for Parkinson disease, head here: FDA Approves Apomorphine Infusion Device SPN-830 as New Parkinson Treatment Episode Breakdown: 1:05 – Significance of SPN-830's approval and impact on PD community 3:10 – Considerations for treatment selection with SPN-830, conversations with families 5:50 – Neurology News Minute 7:50 – TOLEDO study, supportive efficacy and safety data for SPN-830 11:45 – Expanding PD treatment options and overviewing recent progress in research The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Vutrisiran for ATTR-CM, Expanding Indication in Amyloidosis FDA Accepts Regulatory Submission for BTK Inhibitor Tolebrutinib for Non-Relapsing Secondary Progressive MS Simufilam Fails to Meet Key End Points in Phase 3 Alzheimer Disease Trial Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, "Advancing Neuromuscular Care and Research," former FDA commissioner Robert Califf, MD, explored the urgent need for sustained NIH funding to advance basic and translational research, including gene editing, assistive devices, and the integration of artificial intelligence. He emphasized the collaborative spirit of the neuromuscular community, where shared technologies and insights can address multiple disorders. The conversation, which took place at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, highlighted critical challenges, such as clinician shortages, healthcare system strain, and the need for post-market learning to manage uncertainties with emerging therapies like gene therapy. Additionally, he called for innovative funding solutions to address the high costs of treatment, ensure equitable access, and maintain progress in neuromuscular care while fostering long-term health equity. Looking for more epilepsy discussion? Check out the NeurologyLive® neuromuscular clinical focus page. Episode Breakdown: 1:05 – Funding priorities for neuromuscular research and NIH support 1:55 – Role of gene editing, assistive devices, and AI in care 4:50 – Collaboration and togetherness of the neuromuscular community 6:35 – Considerations as new therapeutics emerge for neuromuscular disorders 8:05 – Funding solutions for equitable care Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we cover the recent approval of Genentech's tenecteplase, marketed as TNKase, as a new treatment for adults with acute ischemic stroke (AIS). The thrombolytic medicine is an intravenous tissue plasminogen activator that is clot-dissolving, administered as a single 5-second intravenous bolus. Tenecteplase is only the second such approval for this indication, following the 2015 approval of alteplase (Activase; Genentech); however, tenecteplase is considered faster and more simply administered than alteplase. To better understand the implications of this approval, NeurologyLive sat down with stroke expert Bijoy Menon, MD, MSc, FRCPC, a professor of neurology at the University of Calgary. In the interview, Menon talked about the downstream impacts of having another approved therapy, how tenecteplase differs from other thrombolytic agents, and the efficacy and safety that supported its approval. Furthermore, he provided commentary on how the approval continues to chip away toward the idea of precision medicine and personalizing treatments for patients with AIS.  For more of NeurologyLive's coverage of sodium oxybate's (Lumryz) expanded indication, head here: FDA Approves Tenecteplase for Acute Ischemic Stroke Episode Breakdown: 1:40 – Immediate reaction and significance of tenecteplase approval 5:30 – Safety considerations and administration when prescribing tenecteplase 10:20 – Supportive efficacy and safety, phase 3 AcT trial, and other supplementary studies 14:50 – Advancing precision medicine, treatment personalization with new approval Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Sonal Bhatia, MD, FACNS, epilepsy expert and director of the pediatric epilepsy monitoring unit (EMU) at the Medical University of South Carolina, discussed Purple Day, celebrated on March 25th, and the importance of raising awareness for patients with epilepsy, a common condition affecting one in 26 individuals. She discussed the stigma faced by patients with epilepsy, especially in parts of the world outside the United States. Bhatia also discussed some of the recent advances in epilepsy care in the past decade, highlighting areas like improved surgical approaches, dietary therapies, and the potential of gene therapy for various disorders. In addition, she discussed changes in elderly epilepsy care, as well as the importance of transitioning care from pediatrics to adults and the need for a longitudinal model of care. Furthermore, she spoke on epilepsy monitoring units, the challenges institutions face, and the critical need for EEG technology and for more EEG technologists and nursing school graduates.  Looking for more epilepsy discussion? Check out the NeurologyLive® Epilepsy clinical focus page. Episode Breakdown: 1:10 – Significance of Purple Day, raising awareness on epilepsy stigma 5:00 – Advances in epilepsy care, including availability of more anti-seizure medications and genetic insights 8:45 – Neurology News Minute 11:10 – Challenges in elderly epilepsy care and the need for more standardized transition models from pediatrics to adulthood 15:20 – Optimizing epilepsy monitoring units and dealing with staff and equipment shortages 20:50 – Closing thoughts The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Medtronic's Adaptive Deep Brain Stimulation for Parkinson Disease FDA Approves Tenecteplase for Acute Ischemic Stroke FDA Approves Expanded Use of Eculizumab for Pediatric Myasthenia Gravis Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Julie Flygare, JD, president and chief executive officer of Project Sleep, shared her personal and professional insights into the critical role of social connections for patients living with narcolepsy and idiopathic hypersomnia. Drawing from her journey with narcolepsy, Flygare highlighted the challenges of isolation at diagnosis and the transformative power of peer support and advocacy. She delved into survey findings that underscore the rarity of social connections among patients and their impact on adjustment and well-being. Additionally, Flygare offered actionable recommendations for clinicians to integrate social support into practice, emphasizing the value of patient advocacy organizations and fostering meaningful connections. Above all, her inspiring perspective shed light on the intersection of community, research, and clinical care in sleep health. Looking for more narcolepsy discussion? Check out the NeurologyLive® Narcolepsy clinical focus page. Episode Breakdown: 1:00 – Challenges and importance of social connections 5:45 – Building a thriving community and research impact 7:30 – Insights from patient surveys on social support 10:30 – Neurology News Minute 12:25 – Recommendations for clinicians on social support The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Mirdametinib for NF1-Associated Plexiform Neurofibromas in Adults and Children FDA Approves Tablet Formulation of Risdiplam for Spinal Muscular Atrophy Solid Reports Positive Data for SGT-003 Gene Therapy in Phase 1/2 Trial of Duchenne Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Veronique Miron, PhD, The John David Eaton Chair in Multiple Sclerosis at the University of Toronto, gave a clinical overview of the 2025 ACTRIMS Forum, which is held February 27-March 1, in West Palm Beach, Florida. Miron, an expert in the field, spoke on the reasons behind this year's theme of "Making Connections," as well as some of the most notable and innovative sessions attending clinicians should keep their eyes on. Additionally, she spoke on how the curriculum at this year's meeting aligns with the most recent MS McDonald Diagnostic Criteria that was presented at ECTRIMS 2024. Miron also spoke on the different seminars that cover drug development, innovative new treatment approaches, and Bruton Tyrosine Kinase (BTK) inhibitors. Furthermore, she provided commentary on how this year's meeting differs from previous iterations of ACTRIMS, highlighting some of the changes to the agenda and the advances in MS research. Looking for more multiple sclerosis discussion? Check out the NeurologyLive® Multiple sclerosis clinical focus page. Episode Breakdown: 1:10 – Reasons behind "Making Connections" theme  2:50 – Overviewing key unique sessions and forums from the meeting 5:10 – Aligning curriculum with 2024 MS McDonald Criteria 7:00 – Neurology News Minute 9:00 – Drug development topics from ACTRIMS 2025 12:20 – Differences between ACTRIMS 2025 vs ACTRIMS 2024 The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Axsome Therapeutics' AXS-07 for Migraine Treatment FDA Approves Vertex Pharmaceuticals' Suzetrigine for Acute Pain Management FDA Approves Apomorphine Infusion Device SPN-830 as New Parkinson Treatment Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, epilepsy expert Depobam Samanta, MD, MS, FAAP, FAES, sat down to discuss a recently published guidance paper from members of the Pediatric Epilepsy Research Consortium that provides recommendations for neuromodulation approaches to treat Lennox-Gastaut syndrome (LGS). Samanta, medical director of the Arkansas Children's Comprehensive Epilepsy Program, gave a clinical overview of the paper, highlighting the various neuromodulatory devices and their use, and the ways to improve treatment selection and personalization. Additionally, he talked about some of the potential complications with these devices as well as initiation and titration strategies for easy initiation. Furthermore, he spoke about ways to take neuromodulation to the next level, giving insight on creative trial designs, overcoming sham-controlled studies, and the necessary research to expand these approaches.  Looking for more epilepsy discussion? Check out the NeurologyLive® Epilepsy clinical focus page. Episode Breakdown: 1:05 – Overview of the published guidance and reasons behind the paper  5:15 – Patient selection for neuromodulation and tailoring treatments by patient and preference 8:25 – Overcoming complications with neuromodulation devices  11:25 – Multidisciplinary team necessary for successful implementation of neuromodulation 13:15 – Neurology News Minute 15:15 – Initiation and titration strategies for vagus nerve stimulation, deep brain stimulation, and resective surgery 18:30 – Next steps in research and expanding neuromodulation for patients with LGS 21:10 – Finding creative trial designs to test neuromodulation devices, combination approaches The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Accepts BLA for Subcutaneous Autoinjector Formulation of Lecanemab Cell Therapy Bemdaneprocel Advances to Phases 3 Registrational Trial in Parkinson Disease Real-World Study Highlights Positive Treatment Benefits of Tofersen on ALS Disease Progression, Function Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Russ Lebovitz, MD, PhD, chief executive officer and cofounder of Amprion, discussed the company's SAAmplify-aSYN biomarker test, a first-in-class qualitative laboratory developed test and the only seed amplification assay available to aid in the diagnosis of synucleinopathies such as Parkinson disease (PD) and multiple system atrophy (MSA). Lebovitz provided insight on the new technology and its remarkable accuracy in identifying underling a-synuclein pathology using fluorescence changes. He gave a complete overview of the notable study published in The Lancet Neurology that further tested and validated the assay among a heterogenous group of synucleinopathies. Furthermore, he provided clinical context on the feasibility of the assay, the potential for clinical use, and the continued validation needed.  Looking for more movement disorder discussion? Check out the NeurologyLive® Movement disorder clinical focus page. Episode Breakdown: 1:05 – Overviewing mechanistic function of SAAmplify-aSYN biomarker test, its purpose, and how it came about 7:50 – Results from the published study; ways the fluorescence-based amplification method could distinguish MSA from PD and Lewy body dementia 16:25 – Neurology News Minute 18:30 – Therapeutic feasibility of the assay in clinical settings and the next steps in validation The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Axsome to Submit NDA for AXS-05 in Alzheimer Agitation Following Positive Phase 3 Trials FDA Clears IND for Trial Assessing Gene Therapy SGT-212 in Friedreich Ataxia FDA Grants Fast Track Designation to Anti-Tau Therapy Posdinemab Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this special episode, we've compiled some of the most compelling conversations from Mind Moments podcast episodes throughout 2024. These discussions spotlight groundbreaking research and advancements in neurology that are propelling the field forward. The NeurologyLive team also explored the impact of several newly FDA-approved therapies, offering clinicians valuable insights into what these treatments mean for patient care and their practical use in the clinic. Featured in this week's episode, in order of appearance, are: Daniel Ontaneda, MD, PhD, a staff member of the Cleveland Clinic Mellen Center for Multiple Sclerosis, who gave an overview on the new 2024 McDonald criteria to diagnose multiple sclerosis (MS), which was presented at the 40th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS). Check out the full episode here: https://www.neurologylive.com/view/episode-125-understanding-major-changes-new-mcdonald-criteria-multiple-sclerosis Ian Kremer, executive director of the leaders Engaged on Alzheimer's Disease (LEAD) coalition, on the changing and exciting advances in Alzheimer disease therapeutics, as well as the discontinuation of aducanumab, the first approved antiamyloid therapy. Check out the full episode here: https://www.neurologylive.com/view/episode-113-lessons-learned-alzheimer-drug-development Andy Berkowski, MD, PhD, vice chair of the American Academy of Sleep Medicine's Practice Guidelines Task Force, discusses the newly revised guidelines for restless legs syndrome, including the latest updates in literature and changes to clinical care. Check out the full episode here: https://www.neurologylive.com/view/episode-129-implications-2024-aasm-guidelines-restless-legs-syndrome Sameea Husain-Wilson, DO, a movement disorder specialist at the Marcus Neuroscience Institute of Baptist Health, provided clinical perspective on the use of the Syn-One diagnostic test for patients with Parkinson disease.Check out the full episode here: https://www.neurologylive.com/view/episode-119-utilizing-the-syn-one-test-to-diagnose-parkinson-disease Jonathan Parker, MD, PhD, an assistant professor of neurosurgery at Mayo Clinic Arizona, spoke at the 2024 American Epilepsy Society Annual Meeting on cell therapy approaches for epilepsy, the challenges the clinical community faces, and the outlook for the future. Check out the full episode here: https://www.neurologylive.com/view/episode-130-promise-behind-cell-therapy-approaches-epilepsy Lawrence Robinson, MD, a senior scientist at Sunnybrook Research Institute, gives insight on educating the next generation of neuromuscular and electrodiagnostic (EDX) practitioners, and the major differences in how this generation of medical students learn relative to previous ones. Check out the full episode here: https://www.neurologylive.com/view/episode-127-adapting-neuromuscular-electrodiagnostic-medicine-education-modern-learners Want more Mind Moments episodes? Click here for all of NeurologyLive®'s podcast episodes. Episode Breakdown: 1:05 – Ontaneda on the incorporation of new biomarkers into the 2024 McDonald Criteria for multiple sclerosis. 7:35 – Kremer on the lessons learned from drug development, including antiamyloid treatments, and initial reaction to the discontinuation of aducanumab for AD.  10:30 – Berkowski on the outlook of treating and preventing restless legs syndrome going forward using the newly updated American Academy of Sleep Medicine treatment guidelines.  13:10 – Husain-Wilson on the function of the Syn-One test for Parkinson disease, its applicability, and how it may distinguish patients with PD from other synucleinopathies. 18:40 – Parker on the promise and thought process behind stem cell approaches for drug-resistant epilepsy, including the different types of cell methods being tested.  21:45 – Robinson on the distinct differences in learning styles based on generation, the importance of adaptive teaching, and the new approaches educators in neuromuscular and electrodiagnostic medicine can take.  Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Jonathan Parker, MD, PhD, an assistant professor of neurosurgery at Mayo Clinic Arizona, spoke about the emerging commotion around cell therapies as a way to treat patients with epilepsy. The discussion, which took place at the 2024 American Epilepsy Society (AES) Annual Meeting in Los Angeles, California, covers the thought process behind this approach and why it may hold greater advantages over other traditional surgeries that result in detrimental cognitive effects. Parker, director of the Device-Based Neuroelectronics Lab, spoke on the different types of cell therapies currently in development, the limitations and challenges associated with these medications, and the need to create innovative trials to appropriately test them. Furthermore, Parker spoke on the therapeutic pipeline of cell therapies for epilepsy, including his experience leading a study site for a first-in-human trial of an investigational agent NRTX-1001, a product derived from human pluripotent stem cells. Looking for more epilepsy discussion? Check out the NeurologyLive® Epilepsy clinical focus page. Episode Breakdown: 1:00 – Idea behind cell therapy to treat epilepsy 4:05 – Current state of stem cell therapies for neurological disorders, focusing on Parkinson disease, stroke, and epilepsy 8:05 – NTE001 study of NRTX-1001, an investigational human embryonic stem cell product 11:45 – Neurology News Minute 13:50 – Unanswered questions and safety concerns with cell therapies; challenges with clinical trial design and the need for more innovative trials 17:35 – Misconceptions or gaps in understanding about cell therapies for epilepsy 20:05 – Unique challenges and potential of stem cell therapies for epilepsy, particularly in younger patients; patient motivation and hope The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Anavex Submits Marketing Authorization Application for Blarcamesine in Alzheimer Disease in the EU Testing Begins for RELIEV-CM2 Study of ShiraTronics Neuromodulation Device in Chronic Migraine STK-001 Gains FDA Breakthrough Designation as Potential Disease-Modifying Treatment for Dravet Syndrome Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Andy Berkowski, MD, PhD, founder of Relax Health, sat down to discuss the American Academy of Sleep Medicine's (AASM) recently published guideline update for the treatment of restless legs syndrome (RLS). Berkowski, who serves as a vice chair of the AASM's Practice Guidelines Task Force, provided clinical insight on the reasons behind the guidelines, pointing to the vast research advances and literature updates since its last iteration in 2012. Additionally, he touched on the greatest changes to the guidelines, noting things like the shift away from dopaminergic agents, the importance of iron in RLS pathophysiology, and the use of alpha-2-delta ligands and intravenous iron as first-line treatments. Berkowski also shared thoughts on how the guidelines impact care for patients of all ages, sexes, and RLS subtypes, as well as some of the more troubling parts of the guidelines to write and areas that were left unanswered. Furthermore, he gave his thoughts on how these guidelines will transform care going forward, including accelerating conversations on preventing RLS in many cases in the near future. Looking for more epilepsy discussion? Check out the NeurologyLive® Sleep disorders clinical focus page. Episode Breakdown: 1:00 – Reasons behind new guidelines, progress in clinical research 2:50 – Overview of the greatest changes to the guidelines and what treating physicians should key in on 11:25 – Neurology News Minute 13:40 – How the guidelines address management based on age, sex, and RLS subtype 16:30 – Challenges when drafting the guidelines and the unanswered questions that remain 20:10 – A promising future for treating, managing, and preventing RLS The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Neurogene Reports Serious Adverse Event in Phase 1/2 Rett Study of Gene Therapy NGN-401 Huntington Agent SAGE-718 to be Discontinued Following Disappointing Phase 2 DIMENSION Trial Results Alzheimer Agent Simufilam Fails to Meet Primary End Point in Phase 3 Study Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Lara Jehi, MD, MHCDS, an epilepsy specialist and Cleveland Clinic's Chief Research and Information Officer, sat down to discuss a recently published study that explored using machine learning algorithms to predict seizure control after epilepsy surgery. In the interview, Jehi explained the unique aspects of the study design, emphasizing the importance of a large, well-characterized patient cohort with consistent follow-up and the choice of scalp EEG—a commonly used, non-invasive test in epilepsy care—as the data source. In addition, Jehi touched on the use of AutoML to streamline the process, enabling efficient identification of the top-performing algorithms and enhancing the model's predictive accuracy. Furthermore, she spoke on the team needed to properly implement machine learning techniques for neurosurgery, while providing recommendations for other institutions interested in pursuing these types of approaches. Looking for more epilepsy discussion? Check out the NeurologyLive® epilepsy clinical focus page. Episode Breakdown: 1:00 – Background on various machine learning approaches for epilepsy research 3:20 – Study details, findings, and notable takeaways 8:20 – Neurology News Minute 10:20 – Novelty in using scalp EEG and its global application 15:30 – Team personnel needed for proper implementation of machine learning techniques in epilepsy surgery The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Accepts Resubmitted NDA for Ataluren in Nonsense Duchenne Muscular Dystrophy FDA Places Clinical Hold on Epilepsy Agent RAP-219 for Diabetic Peripheral Neuropathic Pain First-Ever CRISPR/Cas13-RNA Editing Therapy to be Tested in Phase 1 Study of Age-Related Macular Degeneration Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Lawrence Robinson, MD, a senior scientist at Sunnybrook Research Institute, sat down to discuss his presentation from the 2024 American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) annual meeting, focused on educating the next generation of neuromuscular and electrodiagnostic (EDX) practitioners. Robinson gave an overview of his presentation, why this was a topic of interest, and the major differences in how this generation of medical students learn relative to previous ones. In addition, he discussed adapting to new learning styles, the benefits of flipped classrooms, and the impact of technology on education and practice. Furthermore, he touched upon the personal connections and humor in teaching, as well as ways to improve critical evaluation skills as a learner. Looking for more neuromuscular discussion? Check out the NeurologyLive® neuromuscular clinical focus page. Episode Breakdown: 1:05 – Overviewing and background on AANEM presentation 3:15 – Challenges with educating next generation of neuromuscular and EDX practitioners 4:40 – Areas of opportunity and growth for these next generation learners 5:50 – Neurology News Minute 8:00 – Novelty and advantages of flipped classroom approaches to teaching 10:10 – Future expected changes to neuromuscular care 11:55 – Final thoughts on care on NM and EDX education The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Phase 3 ENSURE Program of Vidofludimus Calcium Continues Following Positive Futility Analysis Gene Therapy FLT201 Shows Promise in Early-Stage Study of Gaucher Disease Risk of ARIA-E in Donanemab Attenuated Through New Enhanced Titration Method of Delivery Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we cover the recent approval of Avadel's sodium oxybate formulation (Lumryz) as a treatment for pediatric patients with narcolepsy aged 7 years and older. The new indication expands on its previous one, granted in May 2023, which included the the treatment of cataplexy or excessive daytime sleepiness in adults with narcolepsy. It's initial approval was based on data from the phase 3 REST-ON trial (NCT02720744), a large-scale study in which the therapy met all 3 of its primary end points of change from baseline in mean sleep latency on the Maintenance of Wakefulness test, Clinical Global Impression Improvement, and weekly cataplexy attacks. Following its new indication, NeurologyLive sat down with Anne Marie Morse, DO, FAASM, a pediatric sleep disorders expert, to discuss the significance of the new approval. Morse, director of Child Neurology and Pediatric Sleep Medicine at Geisinger Janet Weis Children's Hospital as well as the program director for Child Neurology Residency Program, provided commentary on the clinical considerations behind this once-nightly formulation of sodium oxybate, and how it should be used going forward. For more of NeurologyLive's coverage of sodium oxybate's (Lumryz) expanded indication, head here: FDA Approves Avadel's Sodium Oxybate for Cataplexy or Excessive Daytime Sleepiness in Pedatric Narcolepsy Episode Breakdown: 2:10 – Immediate reaction and significance  4:10 – Considerations and caution with prescribing sodium oxybate 8:20 – Closing remarks and the need to utilize highly effective treatments for narcolepsy Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Daniel Claaseen, MD, MS, a professor of neurology and chief of the Behavioral and Cognitive Neurology Division at Vanderbilt University Medical Center, offered his insight on phase 1/2 data that was recently presented at the International Parkinson and Movement Disorder Society (MDS) Congress on ATH434, an investigational drug in development from Alterity Therapeutics for multiple system atrophy (MSA). He dove into the therapy's mechanism of action and the currently known safety profile and considerations for its use, as well as the next steps in advancing care for patients with MSA as a whole. Looking for more movement disorders discussion? Check out the NeurologyLive® movement disorder clinical focus page. Episode Breakdown: 1:10 – Overviewing the conduct of the phase 1/2 studies 3:00 – Notable findings from data presented at MDS 4:30 – Mechanism of action behind ATH434 8:20 – Neurology News Minute 10:20 – Next steps in ATH434's development 11:15 – Current unmet needs for patients with MSA The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Removes Partial Hold for Myotonic Dystrophy Agent AOC 1001 FDA Approves Avadel's Sodium Oxybate for Cataplexy or Excessive Daytime Sleepiness in Pedatric Narcolepsy FDA Approves AbbVie's 24-Hour Foscarbidopa/Foslevodopa Pump for Advanced Parkinson Disease Treatment Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Daniel Ontaneda, MD, PhD, a staff member of the Cleveland Clinic Mellen Center for Multiple Sclerosis, provided an in-depth overview on the newly announced McDonald criteria to diagnose multiple sclerosis (MS). Ontaneda, who also serves as an assistant professor of neurology, discussed the most notable changes to the criteria and how it may help towards improving rates of misdiagnosis and missed diagnosis. He spoke on the incorporation of new biomarkers, such as central vein sign and paramagnetic rim lesions, and the ability to diagnose MS in stages before clinical presentation shows. Furthermore, Ontaneda gave some perspective on the areas of the new criteria that will take some time to adjust to, as well as how these criteria may change the philosophical perspective of the disease as a whole.  Looking for more Multiple sclerosis discussion? Check out the NeurologyLive® multiple sclerosis clinical focus page. Episode Breakdown: 1:10 – Focus and direction of new diagnostic criteria 3:00 – Overviewing specific changes to McDonald criteria 10:50 – Approach to diagnosing pre-MS; treating radiologically isolated syndrome earlier 12:55 – Neurology News Minute 15:10 – Incorporation of new biomarkers, central vein sign, paramagnetic rim lesions, OCT 21:45 – Unanswered questions/challenges left out of the diagnostic criteria The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Potential Regulatory Submission for Inebilizumab in Myasthenia Gravis Following Positive Phase 3 MINT Data Parkinson Agent Tavapadon Meets Primary and Secondary End Points as Monotherapy in Phase 3 TEMPO-1 Trial Sage Therapeutics and Biogen Officially Announce Ending of SAGE-324 Program in Essential Tremor Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Joanne Donovan, MD, PhD, chief medical officer at Edgewise Therapeutics, sat down to discuss the clinical program of EDG-5506, an investigational drug in development for Becker muscular dystrophy, and the recent progress in treating and understanding Becker muscular dystrophy. She spoke on the mechanism of action of the drug, also known as sevasemten, and why the company believes it can be therapeutically beneficial for this patient population. Furthermore, Donovan spoke on the previous failures in drug development for Becker, advances in biomarker detection, and how other trials paved the way for the company's phase 2 study and open label extension, dubbed CANYON (NCT05291091) and GRAND CANYON. Furthermore, she spoke on the ways the clinical community has tried to improve the quality of life for patients with Becker, focusing on approaches and tactics that curve cardiac issues commonly seen in this group.  Looking for more Neuromuscular discussion? Check out the NeurologyLive® neuromuscular clinical focus page. Episode Breakdown: 1:15 – Overview of CANYON study and extension phase  3:50 – Mechanism of action of EDG-5506 6:30 – How previous trial experiences shaped CANYON  10:10 – Neurology News Minute 11:50 – Ongoing challenges with treating and testing drugs for Becker 15:25 – Safety profile of EDG-5506 16:45 – Ways to tackle cardiac issues in Becker The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Digital Therapeutic CT-132 Meets Primary End Point in Phase 3 Study of Preventive Migraine FDA Supports Alpha-Synuclein Seed Amplification Assay Biomarker for Clinical Trials in Parkinson Disease FDA Approves Subcutaneous Formulation of Ocrelizumab for Relapsing and Progressive Multiple Sclerosis Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, epilepsy expert Imad Najm, MD, PhD, sat down to discuss the upcoming Cleveland Clinic Epilepsy Summit, an educational symposium that takes place September 11-15, in Cleveland, Ohio. Najm, who serves as the director of Cleveland Clinic's Epilepsy Center at the Cleveland Clinic Neurological Institute, spoke about the objectives and goals of the meeting, including updates on sEEG, epilepsy surgery, treatments for drug-resistant epilepsy, and gene therapy. In addition, he provided an overview of the latest advances in epilepsy surgery, genetics, and seizure controlling approaches. Furthermore, he spoke on some of the educational challenges with adjusting to changes in neurosurgery, the potential benefits and limitations of gene therapy, and the future of epilepsy treatment.  Looking for more Epilepsy discussion? Check out the NeurologyLive® Epilepsy clinical focus page. Episode Breakdown: 1:10 – Mission and goals of Epilepsy Summit  3:05 – Overviewing sessions and educational attainment 4:55 – Changes to epilepsy surgery, advances over the years 9:15 – Neurology News Minute 11:50 – Learning curve for understanding new epilepsy surgery tactics 13:50 – Closing thoughts on the upcoming meeting 15:50 – Potential benefits and challenges to gene therapy for epilepsy The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: EMA Accepts Marketing Authorization Application for Huntington Disease Agent Pridopidine BTK Inhibitor Tolebrutinib Slows Disability Progression in Phase 3 HERCULES Study of Non-Relapsing Secondary Progressive MS FDA Accepts Resubmitted New Drug Application for Migraine Treatment AXS-07 Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Sharon Cohen, MD, a behavioral neurologist, sat down to discuss positive data from an ongoing phase 1 study of mivelsiran (Alnylam Pharmaceuticals), the first investigational RNA interference therapeutic targeting amyloid precursor protein for Alzheimer disease (AD). Cohen, who also serves as the medical director of the Toronto Memory Program at the University of Toronto, discussed the potential of RNA therapeutics for treating AD, the unique mechanism of action of mivelsiran, and some of the early promising safety, efficacy, and pharmacokinetic data observed in the phase 1 trial. In addition, Cohen touched upon the idea of how RNA therapeutics could be used in combination with previously approved novel treatments and the benefits mivelsiran brings with no observed amyloid-related imaging abnormalities. Furthermore, the discussion covered some of the potential of this investigational agent, what to expect in the multi-dose part B of the study, and an additional phase 2 study in cerebral amyloid angiopathy.  Looking for more Alzheimer disease/dementia discussion? Check out the NeurologyLive® Alzheimer disease/dementia clinical focus page. Episode Breakdown: 1:15 – Mechanism of action of mivelsiran and how it can be potentially beneficial in AD 4:15 – Growing knowledge of RNA therapeutics and their impact on neurologic conditions 7:00 – Neurology News Minute 10:10 – Phase 1 study data, including efficacy, safety, biomarker, and pharmacokinetic results 19:20 – How mivelsiran fits with other approved therapies for AD; future goals and directions of the drug The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Clears Indapta Therapeutics' IND for Cell Therapy IDP-023 in Progressive Multiple Sclerosis Muscle-Targeting Therapy Apitegromab Effective in Spinal Muscular Atrophy Over 4 Year Period FDA Approves Medtronic's Deep Brain Stimulation Technology for Asleep Capabilities Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Phil Jochelson, therapeutic head for Clinical Development Neuroscience at Jazz Pharmaceuticals, sat down to discuss the phase 4 XYLO study, a randomized controlled study evaluating the effects of switching from high-sodium to low-sodium oxybate on blood pressure in patients with narcolepsy. Jochelson gave some insight regarding the conduct of the trial, including end points, objectives, and patient demographics, as well as some of the benefits to studying blood pressure and cardiovascular risks. In addition, he discussed enhancements to narcolepsy care through low-sodium oxybate, the importance of unique studies like XYLO, and the need to educate clinicians on sodium load and its risks.  Looking for more sleep disorders discussion? Check out the NeurologyLive® sleep disorders clinical focus page. Episode Breakdown: 1:15 – Reasons behind the XYLO study and links between high sodium intake and cardiovascular risk 4:45 – Patient demographics; inclusion and exclusion criterias 6:40 – Neurology News Minute 9:25 – Overview of XYLO and determining what a successful study looks like 15:30 – Improved understanding of the benefits of low-sodium oxybate The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: EU Regulatory Review Adopts Negative Opinion of Lecanemab as Treatment for Alzheimer Disease GLP-1 Agonist Liraglutide Shows Protective Effects on Alzheimer Disease in Phase 2 Trial FDA Approves Carbidopa/Levodopa ER Capsules Formulation IPX203 for Parkinson Disease Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Michael Weiss, chief executive officer of TG Therapeutics, discussed ublituximab (Briumvi), a recently approved anti-CD20 therapy for relapsing multiple sclerosis, and the advantages it brings to patients. Specifically, he talked about the Department of Veteran Affairs' decision to award national contract to ublituximab as the preferred anti-CD20 for this patient population, and how this facilitates greater access to the treatment. In addition, he discussed how the knowledge profile of the agent has grown over time, as well as the additional efforts to gain a better understanding of its efficacy and safety. Furthermore, he spoke on the differences between ublituximab and other approved agents, and why it may be more applicable to certain patients.  Looking for more multiple sclerosis discussion? Check out the NeurologyLive® Multiple sclerosis clinical focus page. Episode Breakdown: 1:05 – Significance of ublituximab awarded national contract as preferred anti-CD20 for relapsing MS 3:30 – Advantages and differences of ublituximab vs approved therapies for MS 11:40 – Neurology News Minute 14:30 – Growing knowledge profile of ublituximab over the years 19:05 – Future plans of ubtlituximab; long-term goals of the therapy The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Eli Lilly Announces Date for Donanemab FDA AdComm Hearing Patient Death Reported in Phase 2 DAYLIGHT Study of Pfizer's Gene Therapy for Duchenne ALS Candidate PrimeC Shows Greater Treatment Effect in High-Risk ALS Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Douglas Wajda, PhD, an assistant professor of neurology at Cleveland State University, discussed a pilot study presented at the 2024 Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting using a functional electrical stimulation approach, Neural Sleeve, to treat gait in MS. Wajda, who has been studying gait in MS for several years, gave clinical insight on the functionality and feasibility of this approach, and the personalized treatment patients can gain from it. He also discussed the pilot study in detail, including the major end points and takeaways from the small group of patients observed.  Looking for more multiple sclerosis discussion? Check out the NeurologyLive® multiple sclerosis clinical focus page. Episode Breakdown: 1:00 – Functionality of Neural Sleeve 2:45 – Feasibility and self-administration of Neural Sleeve 3:50 – Neurology News Minute 5:50 – Greatest clinical takeaways from pilot study 8:15 – Steps in advancing this therapeutic approach 9:45 – Challenges with gait in MS and personalizing treatment methods The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Gene Therapy LX2006 Positively Impacts Cardiac Biomarkers of Friedreich Ataxia, Clinical Data Show Buntanetap Transitioning to Crystal Form Following FDA Go-Ahead Endo Voluntarily Recalls One Lot of Clonazepam Tablets Following Mislabeling Error Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Sameea Husain-Wilson, DO, a movement disorder specialist at the Marcus Neuroscience Institute of Baptist Health, provided clinical perspective on the use of the Syn-One diagnostic test for patients with Parkinson disease (PD). She gave an overview of how the test is utilized in clinic, ways to interpret results, and the right personnel needed to ensure an accurate diagnosis. In addition, she provided clarity on the role of alpha-synuclein in PD, the advances in research in this area, and how the test incorporates this prominent biomarker. Furthermore, Husain-Wilson detailed some of the next steps in further optimizing the test in clinical settings, highlighting the importance of other non-movement disorder specialists who will play a major role in the diagnostic evaluation of future patients with PD. Looking for more movement disorder discussion? Check out the NeurologyLive® movement disorder clinical focus page. Episode Breakdown: 1:10 – Step by step process on how Syn-One test is used; considerations after process is done 3:15 – How the test differentiates PD from other neurodegenerative disorders 9:50 – Neurology News Minute 12:20 – Value and role in assessing alpha-synuclein in PD 14:50 – Ways to further optimize Syn-One going forward The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Eli Lilly's Donanemab for Early Symptomatic Alzheimer Disease Buntanetap Improves Motor, Nonmotor and Cognitive Symptoms of Parkinson Disease in Phase 3 Study Gene Therapy AMT-130 Slows Huntington Disease Progression in Interim Phase 1/2 Trials Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we're covering the recent approval of donanemab as a new treatment for adults with early symptomatic Alzheimer disease (AD). Marketed as Kisunla, donanemab's approval marks the third antiamyloid therapy to get FDA greenlight for early-stage AD, following the controversial approval of aducanumab (Aduhelm; Biogen) in 2021 and lecanemab (Leqembi; Eisai) in 2023. Donanemab, administered as a 350 mg/20 mL once-monthly injection for intravenous infusion, had its approval supported by the phase 3 TRAILBLAZER-ALZ-2 trial (NCT04437511), a large-scale, double-blind, placebo-controlled trial that featured 1736 patients with early-stage AD. Following the approval, NeurologyLive sat down with Joel Salinas, MD, MBA, a behavioral neurologist at NYU Langone and clinical assistant professor in the department of neurology at the NYU Grossman School of Medicine. Salinas, who also serves as the chief medical officer at Isaac Health, discussed the positive impacts of the approval, the importance of patient selection for the medication, and how clinicians should discuss its benefits and harms to patients. In addition, he commented on how approvals like donanemab continue to carry momentum in the AD field going forward.  For more of NeurologyLive's coverage of donanemab's approval, head here: FDA Approves Eli Lilly's Donanemab for Early Symptomatic Alzheimer Disease Episode Breakdown: 2:10 – Positive downstream impacts of donanemab's approval 4:20 – Considerations and caution with prescribing donanemab 6:05 – Salinas on patient-clinician conversations about AD treatments 8:00 – Closing remarks and continued progress in AD field Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Anne Marie Morse, DO, FAAN, a pediatric neurologist and sleep medicine specialist at Geisinger Medical Center, sat down at the 2024 SLEEP Annual Meeting to discuss a poster presentation highlighting a listening analysis that characterized the struggles and unmet needs of people with narcolepsy. in the discussion, Morse outlined some of the major findings, including the rates of missed and misdiagnosis, how cataplexy impacts daily life, and some of the other comorbidities associated with the condition. She spoke on the value of gaining patient perspectives and using their descriptions for their disorders helps in understanding true unmet needs. Furthermore, she spoke on the importance of a strong patient-clinician relationship, the ability to communicate openly, and the steps following diagnosis to ensure effective long-term treatment outcomes.  Looking for more sleep disorders discussion? Check out the NeurologyLive® sleep disorders clinical focus page. Episode Breakdown: 1:10 – Motivations behind conducting listening analysis 3:15 – Major clinical takeaways from study; notable quality of life impairments in narcolepsy 7:00 – Impact of cataplexy and patients' overreaction 9:50 – Neurology News Minute 12:20 – Reasons for missed and misdiagnosis 14:50 – Early tips to pathing patients towards treatment success The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Grants Traditional Approval to Elevidys Gene Therapy for Ambulatory DMD, Accelerated Approval for Nonambulatory Patients FDA Approves Efgartigimod as New Treatment for Chronic Inflammatory Demyelinating Polyneuropathy FDA Approves Pitolisant for Excessive Daytime Sleepiness in Pediatric Narcolepsy Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, we spoke with a variety of specialists in neurology who presented research and gave talks at the 2024 Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting, held May 29-June 2, in Nashville, Tennessee. Those included in this week's episode, in order of appearance, are: Anthony Feinstein, PhD, FRCPC, MBBCh, a professor of psychiatry at the University of Toronto. Brian G. Weinshenker, MD, a professor of neurology at the University of Virginia. Douglas A. Wajda, PhD, an assistant professor of neurology and member of the Health and Human Performance Department in the College of Education at Cleveland State University. Le Hua, MD, director of Clinical Operations and director of the Multiple Sclerosis Program at Cleveland Clinic's Lou Ruvo Center for Brain Health in Las Vegas, Nevada.  Eion P. Flanagan, MB, BCh, a professor of neurology and chief of the Division of Multiple Sclerosis and Autoimmune Neurology at Mayo Clinic; and director of the Autoimmune Neurology Fellowship.  Want more from the 2024 CMSC Annual Meeting? Click here for all of NeurologyLive®'s coverage of CMSC 2024. Episode Breakdown: 1:40 – Feinstein on the difficulties with improving fatigue in multiple sclerosis and the lack of improvement seen from approved disease-modifying therapies. 5:20 – Weinshenker on the key diagnostic aspects of neuromyelitis optica spectrum disorder and how it differs from other similarly presenting autoimmune disorders. 9:40 – Wajda on the use of the Cionic Neural Sleeve, the advantages it holds, and how it may be used to treat gait dysfunction in multiple sclerosis. 12:00 – Hua on the differential diagnosis of pediatric-onset MS vs late-onset MS, and the notable biologic changes observed. 19:30 – Flanagan on the diagnostic pearls for MOG-antibody associated disease and the importance of early recognition of this group.  Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Katherine Peters, PhD, a neurologist and neurooncologist at the Preston Robert Tisch Brain Tumor Center at Duke University provided context on a new analysis from the phase 3 INDIGO trial, a study assessing vorasidenib in patients with mutant isocitrate dehydrogenase (mIDH) 1/2 diffuse gliomas. The conversation, which occurred at the 2024 AAN Annual Meeting, covered the covered the therapeutic potential of this agent and its impacts on quality of life, neurocognition, and seizure control. Peters, an expert in the field, provided insight on the mechanism of action of vorasidenib, the added value behind the exploratory analysis, and the next steps in research. Furthermore, she provided context on the patient sample observed and why these data may hold significant weight going forward.  Looking for more neuromuscular discussion? Check out the NeurologyLive® epilepsy clinical focus page. Episode Breakdown: 1:05 – Mechanism of action of vorasidenib, a mIDH 1/2 inhibitor 2:00 – Overview of exploratory analysis results 4:20 – Significance of new data, how it adds to previous primary and secondary outcomes 5:50 – Neurology News Minute 8:25 – Next steps for the study and use of vorasidenib 10:35 – Remaining unmet needs for patients with diffuse gliomas The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Diazepam Buccal Film's Role in Treating Intermittent Pediatric Seizures, with Michael Rogawski, MD, PhD Ocrelizumab Gains EU CHMP Positive Opinion for Subcutaneous Formulation to Treat MS FDA Approves Sprinkle Formulation of Neurocrine Biosciences' Valbenazine for Tardive Dyskinesia or Huntington disease Chorea Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Justin Klee and Josh Cohen, cofounders and cochief executive officers of Amylyx Pharmaceuticals, sat down at the recently concluded AAN Annual Meeting to discuss the results from the pivotal phase 3 PHOENIX trial (NCT) of AMX0035 (Relyvrio) in patients with amyotrophic lateral sclerosis (ALS). The duo provided clarity on the findings, positive takeaways from the disappointing data, and the lessons learned in ALS drug development. In addition, the two provided commentary on the future plans of the therapy in other tauopathies like progressive supranuclear palsy and neurologic conditions like Wolfram syndrome. Furthermore, Klee and Cohen shared thoughts on the company's drug pipeline, including AMX0114, an antisense oligonucleotide in development for patients with ALS.  Looking for more neuromuscular discussion? Check out the NeurologyLive® Neuromuscular clinical focus page. Episode Breakdown: 1:05 – Decision behind removing AMX0035 from market 3:00 – Lessons from PHOENIX, AMX0035 drug development program 6:45 – Unraveling PHOENIX study data  10:00 – Neurology News Minute 12:10 – Potential of AMX0035 in other tauopathies, neurologic conditions 15:00 – Outlook of antisense oligonucleotide AMX0114 in ALS The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Diazepam Buccal Film's Role in Treating Intermittent Pediatric Seizures, with Michael Rogawski, MD, PhD Ocrelizumab Gains EU CHMP Positive Opinion for Subcutaneous Formulation to Treat MS FDA Approves Sprinkle Formulation of Neurocrine Biosciences' Valbenazine for Tardive Dyskinesia or Huntington disease Chorea Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we're covering the recent approval of diazepam buccal film (Libervant; Aquestive Therapeutics) for the treatment of pediatric patients with intermittent, stereotypic episodes of frequent seizure activity (seizure clusters, acute repetitive seizures). Indicated for those between 2 and 5 years of age, the therapy offers patients a compact, easily administered diazepam formulation. The FDA previously granted tentative approval in August 2022 for Libervant for treatment of these patients with epilepsy 12 years of age and older, with U.S. market access for Libervant for this age group of patients subject to the expiration of the existing orphan drug market exclusivity of a previously FDA approved drug scheduled to expire in January 2027. Following the approval, Michael Rogawski, MD, PhD, a distinguished professor of neurology and pharmacology at the University of California Davis Health Medical Center, provided insight on what the decision means for patients and clinicians. Rogawski gave comment on the significance of having a new administration route for diazepam, the safety and feasibility of diazepam buccal film, and some of the major points of emphasis from its clinical program. For more of NeurologyLive's and Contemporary Pediatrics coverage of diazepam buccal film's approval, head here: FDA approves diazepam for seizure clusters in patients 2 to 5 years Episode Breakdown: 0:20 – Diazepam buccal film approved for intermittent seizures in pediatrics 2:00 – Michael Rogawski, MD, PhD, giving reaction to the approval 4:10 – Flexibility with multiple diazepam administration routes 6:55 – Rogawski on the safety of diazepam buccal film amid high need 12:30 – Notable takeaways from the diazepam buccal film trial program Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Ian Kremer, executive director of the leaders Engaged on Alzheimer's Disease (LEAD) coalition, spoke about several relevant topics regarding Alzheimer care as new novel therapeutics emerge. He talked about the lessons learned from the recent discontinuation of aducanumab, and the potential and limitations lecanemab (Leqembi; Eisai) and donanemab (Eli Lilly) may bring. Additionally, he provided comments about what matters to patients, the perception of the FDA approval process, and what is considered "clinically meaningful." Furthermore, he gave perspective on ways to improve drug development and emphasized the need for policy decisions to be based on scientific evidence and not by sensationalized headlines. Looking for more Alzheimer disease/dementia discussion? Check out the NeurologyLive® Alzheimer disease/dementia clinical focus page. Episode Breakdown: 1:10 – Promise in the Alzheimer field in 2024 4:05 – Ways of improving efficiencies with drug develpment 9:10 – Discontinuation of aducanumab 11:10– Neurology News Minute 13:50 – Lessons learned from aducanumab, antiamyloid therapies  22:10– Conversations between clinicians and patients surrounding expectations/limitations of antiamyloid therapies and available treatments The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Agrees to New Specialized Protocol for Phase 3b Study of ALS Agent NurOwn FDA Approves Alternate Administration Routes for Antiseizure Medication Cenobamate Extended Use of Investigational Agent IPX203 Safe in Parkinson Disease Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Francesco Saccà, MD, PhD, an associate professor of neurology at the University of Naples, discussed an ongoing study assessing the use of dimethyl fumarate, an approved therapy for relapsing multiple sclerosis, in patients with Friedreich ataxia (FA). He spoke on the mechanism of action of the agent and why it can be beneficial in this patient population, as well as how it differs from omaveloxolone (Skyclarys; Biogen), the first approved treatment for FA. In addition, he outlined the study design, the primary end point of frataxin decrease, and what would be considered a "successful" study. Furthermore, he spoke on the reasons behind the trial and what led investigators to this point. Looking for more ataxia discussion? Check out the NeurologyLive® ataxia clinical focus page. Episode Breakdown: 1:05 – Origin of the study 3:00 – Conduct of the study, end points, goals  5:10 – Promising mechanism of action of dimethyl fumarate to treat Friedreich ataxia 7:30 – Questions in clinical impact of changes in frataxin 10:00 – Neurology News Minute 12:40 – What is considered a successful study? 14:55 – Patient inclusion, demographic makeup of the study 16:35 – Timeline of the trial and data readout The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Alexion's Ravulizumab-cwvz for Neuromyelitis Optica Spectrum Disorder Epilepsy Agent STK-001 Demonstrates Disease-Modifying Effects in Early Phase Studies of Dravet Syndrome Eisai Submits sBLA for Monthly Intravenous Maintenance Dosing of Alzheimer Therapy Lecanemab Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we're covering the recent approval of givinostat (Duvyzat; Italfarmaco) for the treatment of Duchenne muscular dystrophy (DMD). The therapy, a proprietary histone deacetylase (HDAC) inhibitor, was approved as the first nonsteroidal drug for patients with all genetic variants of DMD. The supporting data for the approval of givinostat comes from the phase 3 EPIDYS trial (NCT02851797). EPIDYS, a randomized, double-blind, placebo-controlled, multicenter study, included 179 ambulant male individuals who were randomly assigned 2:1 to either oral givinostat or placebo for an 18-month treatment period. Following the approval, Sharon Hesterlee, PhD, executive vice president and chief research officer of the Muscular Dystrophy Association, sat down to discuss the significance of the approval and how it changes the care for patients with DMD. She spoke specifically about the mechanism of action of the therapy, its safety profile, and how it may be used with other agents. In addition, she discussed other related topics on gene therapy and unmet needs for this patient population.  For more of NeurologyLive's coverage of givinostat's approval, head here: FDA Approves Italfarmaco's Givinostat for Duchenne Muscular Dystrophy Episode Breakdown: 0:30 – Givinostat approved for Duchenne muscular dystrophy 2:00 – Sharon Hesterlee, PhD, on the approval's implications 3:35 – Positive downstream effects of the approval 4:50 – Hersterlee on the safety profile of givinostat 5:40 – Promising outlook of the DMD field 6:45 – Overcoming roadblocks involved with gene therapy  8:55 – Remaining unmet needs for patients with Duchenne 10:15 – Closing thoughts on the approval  Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Daniel Ontaneda, MD, an associate professor of neurology at the Cleveland Clinic Lerner College of Medicine, sat down to discuss the recently concluded Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum. Ontaneda, program committee vice-chair of the meeting, provided insight on a number of notable sessions and discussions that highlighted the recent advances in the MS field. In addition, he talked about the need to innovate clinical trials, ways to tackle progressive MS, and whether drug trials are truly assessing patients at the right time points. Furthermore, he discussed multi-stage trials, the potential role of Bruton tyrosine kinase (BTK) inhibitors, and how this year's meeting differs from previous ones.  Looking for more multiple sclerosis discussion? Check out the NeurologyLive® multiple sclerosis clinical focus page. Episode Breakdown: 1:05 – Overviewing sessions from ACTRIMS Forum 13:10 – Innovating clinical trial design 16:15 – 2023 ACTRIMS vs 2024 ACTRIMS 20:20 – Neurology News Minute 23:30 – Testing combination approaches for progressive MS 26:45 – The impact of timing therapeutic interventions  This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA AdComm to Review Investigational Alzheimer Agent Donanemab and Phase 3 Results Amylyx Mulls Over Pulling AMX0035 Following Disappointing Phase 3 PHOENIX Findings FDA Issues Complete Response Letter for Long-Acting Form of Glatiramer Acetate for Relapsing Multiple Sclerosis Phase 2 PROCEED Trial of PACAP-Targeting Antibody Lu AG09222 in Migraine Announced Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Alcibiades Rodriguez, MD, medical director of the Comprehensive Epilepsy Center—Sleep Center at NYU Langone discussed several sleep-related topics, including the diagnosis of rare sleep disorders and hypersomnias. He spoke on the treatment of obstructive sleep apnea, the importance of adherence to medication, and the ways clinicians can help patients stick to their treatment regimen. Furthermore, he talked about the conversations between patients and clinicians to ensure an accurate diagnosis, as well as the emerging research in the sleep disorder field over the coming years. Looking for more sleep disorders discussion? Check out the NeurologyLive® sleep disorders clinical focus page. Episode Breakdown: 1:40 – Conversations needed to differentiate diagnoses 4:30 – Appearance of sleep disorders and overlap 6:50 – Complications with untreated sleep apnea 8:25 – Neurology News Minute 10:55 – Ways to improve adherence to medication 13:50 – Emerging research in the field This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Glatiramer Acetate Depot Demonstrates Sustained Longterm Safety Profile as Potential MS Therapy Phase 3 DAYBREAK Trial Highlights Long-Term Efficacy of Ozanimod for Relapsing Multiple Sclerosis SRP-9001 Improves Duchenne Muscular Dystrophy Disease Trajectory Despite Failing to Meet Primary End Point in Phase 3 EMBARK Trial Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Stephen From, chief executive officer of Aruna Bio, sat down to discuss the company's investigational exosome candidate AB126 and its upcoming phase 1b/2a trial in patients with ischemic stroke. He discussed the mechanism of action of the therapy, the advantages of its anti-inflammatory and neuroprotective properties, and how it will be used with other stroke treatments. Furthermore, he gave insight on the complexities with conducting stroke trials and improving neurodegeneration in poststroke patients.  Looking for more stroke discussion? Check out the NeurologyLive® stroke clinical focus page. Episode Breakdown: 1:00 – Overviewing phase 1b/2a trial 7:05 – Mechanism of action of AB126 11:15 – Neurology News Minute 13:45 – Role of AB126 in stroke treatment landscape 17:50 – Challenges with repairing neurodegeneration in stroke This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: European Commission Approves Omaveloxolone as First Therapy for Friedreich Ataxia AVP-786 Falls Short in Phase 3 Study of Alzheimer Disease Agitation FDA Grants Priority Review to Efficacy Supplement for Sarepta Therapeutics' SRP-9001 Indication Expansion Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Igancio Mata, PhD, sat down to discuss a recently published meta-analysis on the genetic backgrounds of Parkinson disease beyond traditional European populations. Mata, an associate professor in the Genomic Medicine Institute at Cleveland Clinic, provided insight on the newly identified novel loci and how the data adds to previous genome-wide association studies. In addition, he gave thoughts on the possibility of gene therapy and some of the barriers that come with it, as well as the feasibility of incorporating genetic checkpoints into clinical trial inclusion criteria.  Looking for more movement disorder discussion? Check out the NeurologyLive® movement disorder clinical focus page. Episode Breakdown: 1:10 – Meta-analysis overview 3:20 – Significance of results, how they add to the literature 6:50 – Feasibility of incorporating genetic screening into clinical trials 8:40 – Neurology News Minute 11:15 – Realistic expectations and potential with gene therapy 15:45 – Next steps in advancing this research This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Biogen Walks Away From Aducanumab to Prioritize Lecanemab and Alzheimer Disease Pipeline Updated Guidelines Published to Improve Care in Specialized Epilepsy Centers FDA Clears IND for Gene Therapy Candidate ETX101 in Dravet Syndrome Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Orrin Devinsky, MD, director of the NYU Langone Comprehensive Epilepsy Center, provided commentary on a recently published study assessing video evidence of sudden unexplained deaths in toddlers. Devinsky, who also serves as a professor of neurology, discussed the importance behind crib cams, the moments captured prior to the deaths assessed, and the involvement of seizure activity even with no prior history. In addition, he spoke on the abnormal sounds and movements of these children, the ways to advance SUDC research, and the next steps in understanding and relaying the data.  Looking for more epilepsy discussion? Check out the NeurologyLive® epilepsy clinical focus page. Episode Breakdown: 1:10 – Reasons behind studying sudden death in toddlers 3:55 – Notable takeaways from trial results 8:15 – Advancing ways to research SUDC/SUDEP 9:40 – Neurology News Minute 12:05 – Realistic ways to lower convulsive activity prior to sleep  15:05 – What abnormal movements/noises can ellucidate 15:55 – Ways to expand the research further This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Approves Takeda's Immune Globulin Infusion, Hyqvia, for Chronic Inflammatory Demyelinating Polyneuropathy FDA Issues Complete Response Letter for Satsuma's DHE Nasal Powder STS101 to Treat Acute Migraine ALS Agent PrimeC to Advance to Phase 3 Study After Positive PARADIGM Trial Data Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Cathleen Lutz, PhD, vice president of the Rare Disease Translational Center at the Jackson Laboratory, provided comment on a recently published study which unveiled that stathmin-2 loss leads to neurofilament-dependent axonal collapse in patients with amyotrophic lateral sclerosis (ALS). Lutz offered insight on the role stathmin-2 may have in this disease, the questions that still remain, and whether restoration of stathmin-2 may be an attractive therapeutic approach. Furthermore, she spoke on the potential of this protein as a disease-modifying biomarker and whether it may be incorporated in trials in the near future.  Looking for more neuromuscular discussion? Check out the NeurologyLive® neuromuscular clinical focus page. Episode Breakdown: 1:15 – Idea behind studying stathmin-2 2:45 – Overviewing study results 7:05 – Next steps in extending this research 8:40 – Neurology News Minute 11:30 – Stathmin-2's connection with neurofilament light 12:55 – Potential of stathmin-2 as ALS-specific biomarker 14:45 – Future use of stathmin-2 in clinical trials 15:30 – Closing thoughts This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Clears Phase 2 KYSA-7 Study of CAR T-Cell Therapy in Refractory Progressive Multiple Sclerosis Dyne Reports Positive Phase 1/2 Data for Duchenne Agent DYNE-251 Parkinson Gene Therapy AB-1005 Meets Primary End Point in Phase 1b Trial VISIONARY-MS Trial Reports CNM-Au8 Improves Vision and Cognition in Multiple Sclerosis Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, we comprised some of the most interesting conversations from Mind Moments podcast episodes in 2023, highlighting research in neurology that moves the field forward. The NeurologyLive team included several reaction episodes to FDA approved medicines, providing clinicians an overview of what they can expect from these new therapies and how they will be used in clinic going forward. Those included in this week's episode, in order of appearance, are: Sharon Cohen, MD, FRCPC, behavioral neurologist and medical director of the Toronto Memory Program at the University of Toronto, and a trial investigator for Clarity AD, the phase 3 trial of lecanemab. Check out the full episode here: https://www.neurologylive.com/view/special-episode-lecanemab-approved-for-alzheimer-disease Robert A. Hauser, MD, MBA, director of the Parkinson's and Movement Disorders Center at the University of South Florida.Check out the full episode here: https://www.neurologylive.com/view/episode-81-widening-therapeutic-window-parkinson-disease Alberto Espay, MD, PhD, the division director and Research Endowed Chair of the James J. and Joan A. Gardner Family Center for Parkinson's Disease and Movement Disorders, and a professor of clinical neurology and rehabilitation medicine at the University of Cincinnati.Check out the full episode here: https://www.neurologylive.com/view/episode-94-discussion-leqembi-lecanemab-approval-alzheimer-disease Carolina Ferreira Atuesta, MD, MSc, a scientific researcher and clinical data manager at the Icahn School of Medicine at Mount Sinai.Check out the full episode here: https://www.neurologylive.com/view/episode-82-managing-seizures-after-ischemic-stroke Natalie Goedeker, CPNP, a nurse practitioner in neurology in the Neuromuscular Division at Washington University in St Louis.Check out the full episode here: https://www.neurologylive.com/view/special-episode-tofersen-approved-sod1-als Want more Mind Moments episodes? Click here for all of NeurologyLive®'s podcast episodes. Episode Breakdown: 1:20 – Cohen on the safety of lecanemab, including the 3 patient deaths reported in the supportive clinical trial program.  6:25 – Hauser on the clinical advantages of IPX203, a potential new carbidopa/levodopa formulation for patients with Parkinson disease.  9:20 – Espay on tempering expectations of lecanemab and other antiamyloid therapies in the treatment of Alzheimer disease. 13:20 – Atuesta on the lack of standardized methods for monitoring patients with poststroke seizures, including elders. 17:00 – Goedeker on the approval of gene therapy SRP-9001 for patients with Duchenne muscular dystrophy, the integration process, and safety considerations clinicians should be aware of. This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Jennifer Gudeman, senior vice president of medical and clinical affairs for Avadel Pharmaceuticals, sat down to discuss a recently published post-hoc analysis of the phase 3 REST-ON trial of once-nightly sodium oxybate (Lumryz). In the conversation, Gudeman talked about the impact of the drug on both narcolepsy types 1 and 2, the differences between these 2 subtypes, and the ways clinical trials of narcolepsy agents continue to evolve. Furthermore, she spoke on some of the unmet needs for this patient population, including lingering brain fog and overcoming negative stigmas.  Looking for more sleep disorders discussion? Check out the NeurologyLive® narcolepsy clinical focus page. Episode Breakdown: 1:10 – Overview of post-hoc analysis of REST-ON 3:15 – Differences in type 1 and 2 narcolepsy 5:50 – Neurology News Minute 8:15 – Expanding treatment profile of once-nightly sodium oxybate 10:55 – Lingering unmet needs of patients with narcolepsy 11:20 – Realistic timeline of gene therapies for epilepsy 14:00 – Future of narcolepsy research, drug development trials This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Places Clinical Hold on Roche's BTK Inhibitor Fenebrutinib for Multiple Sclerosis ALS Candidate PrimeC Meets Primary Safety and Secondary End Points in Phase 2b PARADIGM Study FDA Clears NeuroOne's OneRF Ablation System for Neurosurgerical Procedures Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, William Gallentine, DO, interim chief of pediatric neurology at Stanford Medicine Children's Health, who discussed several topics related to the advances in pediatric epilepsy, epilepsy surgery, and seizure localization. Gallentine, who also serves as a clinical professor, provided insight on the screening process and eligibility criteria for epilepsy surgery, as well as the importance of lifestyle modifications when treating epilepsy. Furthermore, he touched upon the genetic background of certain epileptic disorders and when the clinical community can expect gene therapies. Looking for more epilepsy discussion? Check out the NeurologyLive® epilepsy clinical focus page. Episode Breakdown: 1:10 – State of pediatric epilepsy 2:15 – Screening potential candidates for epilepsy surgery 5:00 – Advances in seizure localization 7:15 – Neurology News Minute 9:25 – Balance of nonpharmacological approaches 11:20 – Realistic timeline of gene therapies for epilepsy 13:15 – Future plans for research, current unmet needs This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Clears AI-Powered Brain MRI Software Pixyl.Neuro FDA Grants Meeting With BrainStorm Cell Therapeutics on ALS Therapy NurOwn FDA Warns of Serious Adverse Effects for Using Levetiracetam and Clobazam in Epilepsy Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Brett Vaughan, chief executive officer of Cognito Therapeutics, provided insight on the mechanism of action behind the company's gamma sensory stimulation as a potential therapy for patients with Alzheimer disease. He spoke on the positive phase 2 OVERTURE study presented at the 2023 Clinical Trials on Alzheimer's Disease (CTAD) conference, including the notable takeaways from the trial and the safety profile observed. In addition, he commented on how this approach can be used with newer therapies and what the company is looking to achieve in a phase 3 trial. Looking for more Alzheimer disease/dementia discussion? Check out the NeurologyLive® Alzheimer disease/dementia clinical focus page. Episode Breakdown: 1:10 – Overview of phase 2 OVERTURE study 4:45 – Mechanism of action of gamma sensory stimulation  10:00 – Potential therapeutic crossover with emerging treatments 12:30 – Neurology News Minute 14:50 – Feasibility of the stimulation device 18:20– Future plans and planned phase 3 study This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: ATA188 Fails to Meet Primary End Point In Phase 2 EMBOLD Study of Progressive MS Elecsys Neurofilament Light Test Gains Breakthrough Device Designation as Way to Track MS Disease Activity FDA Clears Phase 2 Study of CAR T-Cell Therapy KYV-101 in Myasthenia Gravis Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we're covering the recent approval of vamorolone (Agamree; Santhera Pharmaceuticals) oral suspension 40 mg/mL for the treatment of patients with Duchenne muscular dystrophy (DMD) aged 2 years and older. The therapy is set to be acquired by Catalyst Pharmaceuticals soon, and is a first-in-class dissociative steroid that aims to retain the anti-inflammatory activity of corticosteroids while decreasing the deleterious adverse events (AEs). We spoke with Barry J. Byrne, MD, PhD, professor and associate chair of pediatrics and director of the Powell Gene Therapy Center at the University of Florida, who discussed the recent FDA approval of Santhera Pharmaceuticals investigational agent vamorolone (now marketed as Agamree, and soon to be acquired by Catalyst Pharmaceuticals in North America), its potential to affect the treatment paradigm as a first-in-class dissociative steroid, and important efficacy and safety information from the phase 2b VISION-DMD study (NCT03439670). For more of NeurologyLive's coverage of vamorolone's approval, head here: FDA Approves Vamorolone as a Treatment for Duchenne Muscular Dystrophy Episode Breakdown: 0:30 – Vamorolone approved for Duchenne muscular dystrophy 2:20 – Barry J. Byrne, MD, PhD, on his immediate reaction 3:25 – Safety data on vamorolone 4:00 – Byrne on the importance of bone mineralization in DMD 5:10 – Phase 2b efficacy data from VISION-DMD 5:45 – Byrne with context on the efficacy data 7:05 – Byrne with context on the landscape of DMD therapeutic development and personalization approaches Thanks for listening to the NeurologyLive Mind Moments podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com. REFERENCES 1. Catalyst Pharmaceuticals Reports FDA Approval of AGAMREE® (vamorolone) for Duchenne Muscular Dystrophy Granted to Santhera Pharmaceuticals. News release. Catalyst Pharmaceuticals. October 26, 2023. Accessed October 26, 2023. https://www.globenewswire.com/en/news-release/2023/10/26/2767947/13009/en/Catalyst-Pharmaceuticals-Reports-FDA-Approval-of-AGAMREE-vamorolone-for-Duchenne-Muscular-Dystrophy-Granted-to-Santhera-Pharmaceuticals.html 2. Santhera and ReveraGen announce positive and statistically highly significant topline results with vamorolone in pivotal VISION-DMD study. News release. Santhera. June 1, 2021. Accessed February 1, 2023. https://www.globenewswire.com/news-release/2021/06/01/2239124/0/en/Santhera-and-ReveraGen-Announce-Positive-and-Statistically-Highly-Significant-Topline-Results-with-Vamorolone-in-Pivotal-VISION-DMD-Study.html 3. Dang U, Guglieri M, Clemens PR, et al. Delayed start analysis of efficacy outcomes in placebo to vamorolone crossover participants in VBP15-004. Presented at MDA Clinical and Scientific Conference; March 13-16. Poster 89. 4. Muscular Dystrophy Association-Supported Drug AGAMREE® (vamorolone) Approved for the Treatment of Duchenne Muscular Dystrophy by the FDA. News release. MDA. October 26, 2023. Accessed October 26, 2023. https://www.mda.org/press-releases/mda-supported-drug-agamree-vamorolone-approved-for-the-treatment-of-duchenne-muscular-dystrophy-by-the-fda 5. Guglieri M, Clemens PR, Perlman SJ, et al. Efficacy and safety of vamorolone vs placebo and prednisone among boys with Duchenne muscular dystrophy: a randomized clinical trial. JAMA Neurol. Published online August 29, 2022. doi:10.1001/jamaneurol.2022.2480

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Alfonso Fasano, MD, PhD, professor of neurology at the University of Toronto and staff neurologist at Toronto Western Hospital, who spoke on the topic of neuromodulation in Parkinson disease and the potential targets clinicians should be learning about. He discussed the specific patient profiles who fit for neuromodulation, ways to improve this type of personalized medicine, and the needed research in this patient population going forward.  Looking for more movement disorder discussion? Check out the NeurologyLive® Movement Disorder clinical focus page. Episode Breakdown: 1:10 – Overview of MDS presentation 2:40 – Reasons for complexities with freezing in parkinsonism 4:35 – Aspects of deep brain stimulation care that need improvement 7:00 – Neurology News Minute 9:15 – Promising neuromodulation targets in the brain 12:35 – Finding optimal candidates for specific neurosurgery procedures 18:50 – Future research of neuromodulation in Parkinson disease This episode is brought to you by Medical World News, a streaming channel from MJH Life Sciences®. Check out new content and shows every day, only at medicalworldnews.com. The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: Eisai Plans to Submit BLA for Subcutaneous Lecanemab After Findings Show Promising Results FDA Approves Vamorolone as a Treatment for Duchenne Muscular Dystrophy SRP-9001 Fails to Meet Primary End Point in Phase 3 EMBARK Study Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we're covering the recent approval of zilucoplan (Zilbrysq; UCB Pharma) for the treatment of patients with acetylcholine receptor antibody–positive generalized myasthenia gravis. We spoke with James F. “Chip” Howard, MD, Distinguished Professor of Neuromuscular Disease and professor of neurology and medicine at The University of North Carolina at Chapel Hill School of Medicine, and the lead global investigator of the phase 3 RAISE study (NCT04115293), who discussed the downstream effects of this approval for myasthenia gravis, the available data from clinical studies, and the advantages for patients and physicians. For more of NeurologyLive®'s coverage of zilucoplan's approval, head here: FDA Approves Complement C5 Inhibitor Zilucoplan as Treatment for Myasthenia Gravis Episode Breakdown: 0:30 – Zilucoplan approved for AChR+ myasthenia gravis 1:40 – James F. Howard, MD, on his immediate reaction 3:30 – Safety data from the RAISE trial 4:25 – Howard on the potential advantages of zilucoplan 6:50 – Phase 3 efficacy data for zilucoplan 7:55 – Howard with context on the efficacy and the challenges with therapeutic availability in myasthenia gravis 12:10 – Howard, on the future of therapeutic development Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com. REFERENCES 1. UCB announces US FDA approval of Zilbrysq (zilucoplan) for the treatment of adults with generalized myasthenia gravis. News release. October 17, 2023. Accessed October 17, 2023. https://www.ucb.com/stories-media/Press-Releases/article/UCB-announces-US-FDA-approval-of-ZILBRYSQR-zilucoplan-for-the-treatment-of-adults-with-generalized-myasthenia-gravis 2. Howard JF, Bresch S, Genge A, et al. Safety and efficacy of zilucoplan in patients with generalized myasthia gravis (RAISE): a randomized, double-blind, placebo-controlled, phase 3 study. 2023;22(5):395-406. doi:10.1016/S1474-4422(23)00080-7 3. Genge A, Hussain Y, Kaminski HJ, et al. Safety and tolerability of zilucoplan in RAISE-XT: a multicenter, open-label extension study in patients with myasthenia gravis. Presented at: MDA 2023; February 19-22; Dallas, TX. Abstract 145.