Podcasts about rare and orphan diseases

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Michael Wang, MD For patients with mantle cell lymphoma (MCL) who relapse after BTK inhibitor (BTKi) therapy, treatment decisions can be complex and time sensitive. That's why understanding how tumor biology and risk features can guide selection between immunomodulatory regimens and CAR T-cell therapy is essential. Tune in to hear Dr. Charles Turck speak with Dr. Michael Wang about practical, evidence-based strategies for managing relapsed/refractory MCL. Dr. Wang is a Professor in the Department of Lymphoma and Myeloma in the Department of Stem Cell Transplantation at MD Anderson Cancer Center in Houston, Texas.

Host: Ryan Quigley In a 2025 study investigating salivary metabolites as potential biomarkers in cystic fibrosis, researchers identified distinct metabolic patterns linked with key complications as well as correlations with lung function. In this AudioAbstract, Ryan Quigley discusses how a simple saliva sample could provide clinicians with an accessible, non-invasive tool to stratify patients, monitor therapies, and advance personalized care in cystic fibrosis. This topic was also discussed at the 2025 CHEST Annual Meeting.

Host: Ryan Quigley In a 2025 study investigating salivary metabolites as potential biomarkers in cystic fibrosis, researchers identified distinct metabolic patterns linked with key complications as well as correlations with lung function. In this AudioAbstract, Ryan Quigley discusses how a simple saliva sample could provide clinicians with an accessible, non-invasive tool to stratify patients, monitor therapies, and advance personalized care in cystic fibrosis. This topic was also discussed at the 2025 CHEST Annual Meeting.

Guest: Alexis Leonard With two FDA-approved gene therapies, including the first CRISPR-based treatment, curative strategies for sickle cell disease are continuing to expand the treatment landscape. However, challenges remain in optimizing safety and accessibility, particularly for patients unable to tolerate current myeloablative conditioning. Learn about advancements in ex vivo editing, such as improved stem cell targeting and multiplex editing, and the possibility of in vivo approaches that may help transform sickle cell disease management with Dr. Alexis Leonard, who works in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee.

Guest: David Jacobsohn Gene therapy is redefining the treatment landscape for sickle cell disease, with approaches like gene addition, gene editing, and fetal hemoglobin induction showing promise in reducing or even eliminating symptoms. However, while these therapies offer some advantages over allogeneic stem cell transplant, they also come with significant challenges. In a conversation with Ryan Quigley, Dr. David Jacobsohn discusses considerations for these therapeutic approaches and their potential impacts on care plans for sickle cell disease patients. Dr. Jacobsohn is the Division Chief of the Blood and Marrow Transplantation Program at Children's National Hospital and Full Professor of Pediatrics at the George Washington University in Washington, DC.

Guest: Alexis Leonard CRISPR technology, while relatively new, has shown potential in treating patients with sickle cell disease by increasing fetal hemoglobin. Though this gene therapy comes with safety considerations, refinements and advancements in CRISPR technology can offer new management options for sickle cell disease patients. Learn about the history of CRISPR as a sickle cell disease treatment and the future of this strategy with Dr. Alexis Leonard, who works in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee.

Guest: Alexis Leonard Sickle cell disease management traditionally involves two primary treatment approaches: disease-modifying therapies and curative strategies. While disease-modifying therapies can help reduce complications associated with the disease, they do not correct or fix them, which is why curative strategies are appealing to some patients and physicians. Join Dr. Alexis Leonard as she discusses the current management landscape for sickle cell disease, including disease-modifying treatments, curative strategies, and potential gene therapies. Dr. Leonard works in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee.

Host: Ryan Quigley Neonatal food protein-induced enterocolitis syndrome (N-FPIES) is an underrecognized condition often misdiagnosed as necrotizing enterocolitis, despite distinct clinical and lab features that demand a different treatment approach. In this AudioAbstract, ReachMD's Ryan Quigley explores the early signs, immune pathways, and diagnostic markers of N-FPIES, offering essential insights to help pediatric clinicians identify this condition and initiate appropriate dietary management to avoid unnecessary interventions.

Host: Ryan Quigley Neonatal food protein-induced enterocolitis syndrome (N-FPIES) is an underrecognized condition often misdiagnosed as necrotizing enterocolitis, despite distinct clinical and lab features that demand a different treatment approach. In this AudioAbstract, ReachMD's Ryan Quigley explores the early signs, immune pathways, and diagnostic markers of N-FPIES, offering essential insights to help pediatric clinicians identify this condition and initiate appropriate dietary management to avoid unnecessary interventions.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Jonathon B. Cohen, MD, MS As the therapeutic landscape for follicular lymphoma continues to evolve, CAR T-cell therapy is emerging as a transformative option for select patients with relapsed or high-risk disease. But it also comes with a lot of important considerations, like knowing when to refer and how to manage common adverse events. Joining Dr. Charles Turck to explore how CAR T fits into the broader treatment algorithm for follicular lymphoma is Dr. Jonathan Cohen. Not only is he a Professor in the Department of Hematology and Medical Oncology at the Emory University School of Medicine, but he's also the Co-Director of the Lymphoma Program at the Winship Cancer Institute of Emory University in Atlanta.

Host: Jennifer Caudle, DO Guest: Katie Beadon, MD, MASc, FRCPC Multifocal motor neuropathy (MMN) is a chronic, immune-mediated motor neuropathy that can mimic other neuromuscular disorders1-3 and is one of the few motor neuron disorders that is treatable.4 However, disease severity correlates with the length of time a patient remains untreated, which is why early recognition and treatment initiation is essential for preserving long-term motor function.5-6 Joining Dr. Jennifer Caudle to discuss the diagnosis and management of MMN is Dr. Katie Beadon, Co-Director of St. Paul's Hospital Immunotherapy in Neurology Clinic and an Assistant Clinical Professor at the University of British Columbia in Vancouver. References: Guimarães-Costa R, Bombelli F, Léger JM. Multifocal motor neuropathy. Presse Med. 2013;42(6 Pt 2):e217-24. doi:10.1016/j.lpm.2013.01.057 Beadon K, Guimarães-Costa R, Léger JM. Multifocal motor neuropathy. Curr Opin Neurol. 2018;31(5):559-564. doi:10.1097/WCO.0000000000000605 Vlam L, van der Pol WL, Cats EA, et al. Multifocal motor neuropathy: diagnosis, pathogenesis and treatment strategies. Nat Rev Neurol. 2011;8(1):48-58. doi:10.1038/nrneurol.2011.175 Yeh WZ, Dyck PJ, van den Berg LH, Kiernan MC, Taylor BV. Multifocal motor neuropathy: controversies and priorities. J Neurol Neurosurg Psychiatry. 2020;91(2):140-148. doi:10.1136/jnnp-2019-321532 Cats EA, van der Pol WL, Piepers S, et al. Correlates of outcome and response to IVIg in 88 patients with multifocal motor neuropathy. Neurology. 2010;75(9):818-25. doi:10.1212/WNL.0b013e3181f0738e Van …

Host: Jennifer Caudle, DO Guest: Katie Beadon, MD, MASc, FRCPC Multifocal motor neuropathy (MMN) is a chronic, immune-mediated motor neuropathy that can mimic other neuromuscular disorders1-3 and is one of the few motor neuron disorders that is treatable.4 However, disease severity correlates with the length of time a patient remains untreated, which is why early recognition and treatment initiation is essential for preserving long-term motor function.5-6 Joining Dr. Jennifer Caudle to discuss the diagnosis and management of MMN is Dr. Katie Beadon, Co-Director of St. Paul's Hospital Immunotherapy in Neurology Clinic and an Assistant Clinical Professor at the University of British Columbia in Vancouver. References: Guimarães-Costa R, Bombelli F, Léger JM. Multifocal motor neuropathy. Presse Med. 2013;42(6 Pt 2):e217-24. doi:10.1016/j.lpm.2013.01.057 Beadon K, Guimarães-Costa R, Léger JM. Multifocal motor neuropathy. Curr Opin Neurol. 2018;31(5):559-564. doi:10.1097/WCO.0000000000000605 Vlam L, van der Pol WL, Cats EA, et al. Multifocal motor neuropathy: diagnosis, pathogenesis and treatment strategies. Nat Rev Neurol. 2011;8(1):48-58. doi:10.1038/nrneurol.2011.175 Yeh WZ, Dyck PJ, van den Berg LH, Kiernan MC, Taylor BV. Multifocal motor neuropathy: controversies and priorities. J Neurol Neurosurg Psychiatry. 2020;91(2):140-148. doi:10.1136/jnnp-2019-321532 Cats EA, van der Pol WL, Piepers S, et al. Correlates of outcome and response to IVIg in 88 patients with multifocal motor neuropathy. Neurology. 2010;75(9):818-25. doi:10.1212/WNL.0b013e3181f0738e Van …

Host: Jennifer Caudle, DO Guest: Katie Beadon, MD, MASc, FRCPC Multifocal motor neuropathy (MMN) is a chronic, immune-mediated motor neuropathy that can mimic other neuromuscular disorders1-3 and is one of the few motor neuron disorders that is treatable.4 However, disease severity correlates with the length of time a patient remains untreated, which is why early recognition and treatment initiation is essential for preserving long-term motor function.5-6 Joining Dr. Jennifer Caudle to discuss the diagnosis and management of MMN is Dr. Katie Beadon, Co-Director of St. Paul's Hospital Immunotherapy in Neurology Clinic and an Assistant Clinical Professor at the University of British Columbia in Vancouver. References: Guimarães-Costa R, Bombelli F, Léger JM. Multifocal motor neuropathy. Presse Med. 2013;42(6 Pt 2):e217-24. doi:10.1016/j.lpm.2013.01.057 Beadon K, Guimarães-Costa R, Léger JM. Multifocal motor neuropathy. Curr Opin Neurol. 2018;31(5):559-564. doi:10.1097/WCO.0000000000000605 Vlam L, van der Pol WL, Cats EA, et al. Multifocal motor neuropathy: diagnosis, pathogenesis and treatment strategies. Nat Rev Neurol. 2011;8(1):48-58. doi:10.1038/nrneurol.2011.175 Yeh WZ, Dyck PJ, van den Berg LH, Kiernan MC, Taylor BV. Multifocal motor neuropathy: controversies and priorities. J Neurol Neurosurg Psychiatry. 2020;91(2):140-148. doi:10.1136/jnnp-2019-321532 Cats EA, van der Pol WL, Piepers S, et al. Correlates of outcome and response to IVIg in 88 patients with multifocal motor neuropathy. Neurology. 2010;75(9):818-25. doi:10.1212/WNL.0b013e3181f0738e Van …

Host: Jennifer Caudle, DO Guest: Katie Beadon, MD, MASc, FRCPC Multifocal motor neuropathy (MMN) is a chronic, immune-mediated motor neuropathy that can mimic other neuromuscular disorders1-3 and is one of the few motor neuron disorders that is treatable.4 However, disease severity correlates with the length of time a patient remains untreated, which is why early recognition and treatment initiation is essential for preserving long-term motor function.5-6 Joining Dr. Jennifer Caudle to discuss the diagnosis and management of MMN is Dr. Katie Beadon, Co-Director of St. Paul's Hospital Immunotherapy in Neurology Clinic and an Assistant Clinical Professor at the University of British Columbia in Vancouver. References: Guimarães-Costa R, Bombelli F, Léger JM. Multifocal motor neuropathy. Presse Med. 2013;42(6 Pt 2):e217-24. doi:10.1016/j.lpm.2013.01.057 Beadon K, Guimarães-Costa R, Léger JM. Multifocal motor neuropathy. Curr Opin Neurol. 2018;31(5):559-564. doi:10.1097/WCO.0000000000000605 Vlam L, van der Pol WL, Cats EA, et al. Multifocal motor neuropathy: diagnosis, pathogenesis and treatment strategies. Nat Rev Neurol. 2011;8(1):48-58. doi:10.1038/nrneurol.2011.175 Yeh WZ, Dyck PJ, van den Berg LH, Kiernan MC, Taylor BV. Multifocal motor neuropathy: controversies and priorities. J Neurol Neurosurg Psychiatry. 2020;91(2):140-148. doi:10.1136/jnnp-2019-321532 Cats EA, van der Pol WL, Piepers S, et al. Correlates of outcome and response to IVIg in 88 patients with multifocal motor neuropathy. Neurology. 2010;75(9):818-25. doi:10.1212/WNL.0b013e3181f0738e Van …

CME credits: 1.00 Valid until: 19-09-2026 Claim your CME credit at https://reachmd.com/programs/cme/bronchiectasis-update-diagnostic-innovations-and-therapeutic-frontiers/37233/ Bronchiectasis is complex, often underdiagnosed, and frequently misunderstood. Explore key contributors to disease progression, including the role of neutrophilic inflammation and neutrophilic serine proteases, and learn to distinguish bronchiectasis from other respiratory conditions and assess patient risk factors that impact outcomes. This module covers how pulmonary exacerbations influence disease course and highlights new insights from recent clinical trials. Stay ahead of the curve with updates on emerging therapies that could transform how we manage this challenging disease.=

CME credits: 1.00 Valid until: 19-09-2026 Claim your CME credit at https://reachmd.com/programs/cme/bronchiectasis-update-diagnostic-innovations-and-therapeutic-frontiers/37233/ Bronchiectasis is complex, often underdiagnosed, and frequently misunderstood. Explore key contributors to disease progression, including the role of neutrophilic inflammation and neutrophilic serine proteases, and learn to distinguish bronchiectasis from other respiratory conditions and assess patient risk factors that impact outcomes. This module covers how pulmonary exacerbations influence disease course and highlights new insights from recent clinical trials. Stay ahead of the curve with updates on emerging therapies that could transform how we manage this challenging disease.=

Host: Ashley Baker, MSN, PMHNP Guest: Alyssa Schlenz Guest: Shannon Phillps Families of children with sickle cell disease have noted both an abundance of support and significant obstacles when accessing care, ranging from knowledgeable providers and flexible scheduling to transportation concerns and insurance holdups. Joining Ashley Baker to discuss their recent research on these key facilitators and barriers are Drs. Alyssa Schlenz and Shannon Phillips. Dr. Schlenz is an Associate Professor of Pediatrics at the University of Colorado School of Medicine, and Dr. Phillips is an Associate Professor in the College of Nursing at the Medical University of South Carolina.

Host: Marlene Mimi Maeusli, PhD. A recent Danish population-based study evaluating real-world outcomes of ibrutinib in relapsed or refractory mantle cell lymphoma (MCL) found lower efficacy and tolerability compared to clinical trials, particularly among older patients and those with high-risk disease features. In this AudioAbstract, ReachMD's Dr. Mimi Maeusli explains how this evidence is reshaping our view of ibrutinib's clinical utility.

Host: Marlene Mimi Maeusli, PhD. A recent Danish population-based study evaluating real-world outcomes of ibrutinib in relapsed or refractory mantle cell lymphoma (MCL) found lower efficacy and tolerability compared to clinical trials, particularly among older patients and those with high-risk disease features. In this AudioAbstract, ReachMD's Dr. Mimi Maeusli explains how this evidence is reshaping our view of ibrutinib's clinical utility.

Host: Marlene Mimi Maeusli, PhD. A recent Danish population-based study evaluating real-world outcomes of ibrutinib in relapsed or refractory mantle cell lymphoma (MCL) found lower efficacy and tolerability compared to clinical trials, particularly among older patients and those with high-risk disease features. In this AudioAbstract, ReachMD's Dr. Mimi Maeusli explains how this evidence is reshaping our view of ibrutinib's clinical utility.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Sairah Ahmed, MD CAR T-cell therapies have helped transform the treatment of aggressive lymphomas, but could they also change the game for slower-growing, harder-to-treat diseases like marginal zone lymphoma? Based on new data from the TRANSCEND FL study, liso-cel achieved a 95 percent overall response rate and sustained 24-month progression-free survival in relapsed/refractory marginal zone lymphoma. Here with Dr. Charles Turck to share the key efficacy and safety findings is Dr. Sairah Ahmed, Professor in the Department of Lymphoma and Myeloma and CAR T Program Director at MD Anderson Cancer Center.

Guest: John Cho, MD, PhD, FRCPC Surgery for mesothelioma after radiation therapy (SMART) offers new hope for patients with resectable epithelioid mesothelioma by reversing the traditional treatment sequence. Hear from Dr. John Cho as he discusses the rationale behind this decade-long clinical advancement, which he spoke about at the 2025 World Conference on Lung Cancer. Dr. Cho is a radiation oncologist at Princess Margaret Cancer Centre and an Associate Professor in the Department of Radiation Oncology at the University of Toronto.

Guest: John Cho, MD, PhD, FRCPC While early findings using the SMART protocol for mesothelioma have been encouraging, replication has proven challenging due to steep surgical learning curves, complexities in planning, and skepticism. Dr. John Cho explores why adoption has been limited and what's next for this approach, which he discussed at the 2025 World Conference on Lung Cancer. Dr. Cho is a radiation oncologist at Princess Margaret Cancer Centre and an Associate Professor in the Department of Radiation Oncology at the University of Toronto.

Guest: John Cho, MD, PhD, FRCPC Surgery for mesothelioma after radiation therapy (SMART) offers new hope for patients with resectable epithelioid mesothelioma by reversing the traditional treatment sequence. Hear from Dr. John Cho as he discusses the rationale behind this decade-long clinical advancement, which he spoke about at the 2025 World Conference on Lung Cancer. Dr. Cho is a radiation oncologist at Princess Margaret Cancer Centre and an Associate Professor in the Department of Radiation Oncology at the University of Toronto.

Guest: John Cho, MD, PhD, FRCPC While early findings using the SMART protocol for mesothelioma have been encouraging, replication has proven challenging due to steep surgical learning curves, complexities in planning, and skepticism. Dr. John Cho explores why adoption has been limited and what's next for this approach, which he discussed at the 2025 World Conference on Lung Cancer. Dr. Cho is a radiation oncologist at Princess Margaret Cancer Centre and an Associate Professor in the Department of Radiation Oncology at the University of Toronto.

Guest: John Cho, MD, PhD, FRCPC While early findings using the SMART protocol for mesothelioma have been encouraging, replication has proven challenging due to steep surgical learning curves, complexities in planning, and skepticism. Dr. John Cho explores why adoption has been limited and what's next for this approach, which he discussed at the 2025 World Conference on Lung Cancer. Dr. Cho is a radiation oncologist at Princess Margaret Cancer Centre and an Associate Professor in the Department of Radiation Oncology at the University of Toronto.

Guest: John Cho, MD, PhD, FRCPC Surgery for mesothelioma after radiation therapy (SMART) offers new hope for patients with resectable epithelioid mesothelioma by reversing the traditional treatment sequence. Hear from Dr. John Cho as he discusses the rationale behind this decade-long clinical advancement, which he spoke about at the 2025 World Conference on Lung Cancer. Dr. Cho is a radiation oncologist at Princess Margaret Cancer Centre and an Associate Professor in the Department of Radiation Oncology at the University of Toronto.

Fibrodysplasia ossificans progressiva (FOP) is a severely disabling myopathy in which extraskeletal bone forms and accumulates over time within muscles and soft tissues.1,2 Here's what we know so far about the mechanisms of heterotopic ossification in patients with FOP. References: Kaplan FS et al. Best Pract Res Clin Rheumatol 2008;22:191–205. Pignolo RJ et al. Orphanet J Rare Dis 2011;6:80. © 2025 Ipsen Biopharmaceuticals Inc. All rights reserved. ALLSC-US-001927 | August 2025

Guest: Mateo Mejia Saldarriaga, M.D. Mateo Mejia Saldarriaga, M.D., a hematologist/oncologist at NewYork-Presbyterian and Weill Cornell Medicine, explains how he and his team conducted a retrospective study that identified a biomarker to enhance treatment planning for BCMA CAR T-cell therapy in multiple myeloma. By measuring absolute lymphocyte count (ALC) through a routine CBC 15 days after a CAR T-cell therapy injection, they found patients with an ALC > 1,000 had a median progression-free survival (PFS) of 30 months whereas patients with an ALC ≤ 500 had a median PFS of 6 months. This new biomarker is now being leveraged to help doctors predict whether a patient is benefiting from treatment in as early as 15 days. © 2025 NewYork-Presbyterian

CME credits: 1.00 Valid until: 31-03-2026 Claim your CME credit at https://reachmd.com/programs/cme/identifying-a-key-cause-of-hard-to-control-diabetes/36543/ The diagnosis of hypercortisolism is often delayed or missed, especially in primary care settings, leading to detrimental consequences for patients, such as unnecessary morbidity and mortality. Recent data suggest that the prevalence of hypercortisolism is far higher than previously estimated. Clinicians need to be aware of optimal approaches for identifying, diagnosing, and treating hypercortisolism. New data are emerging for hypercortisolism treatments that can improve outcomes in patients with hypercortisolism and hard-to-control type 2 diabetes. =

CME credits: 1.00 Valid until: 31-03-2026 Claim your CME credit at https://reachmd.com/programs/cme/identifying-a-key-cause-of-hard-to-control-diabetes/36543/ The diagnosis of hypercortisolism is often delayed or missed, especially in primary care settings, leading to detrimental consequences for patients, such as unnecessary morbidity and mortality. Recent data suggest that the prevalence of hypercortisolism is far higher than previously estimated. Clinicians need to be aware of optimal approaches for identifying, diagnosing, and treating hypercortisolism. New data are emerging for hypercortisolism treatments that can improve outcomes in patients with hypercortisolism and hard-to-control type 2 diabetes. =

Guest: Abiodun Ologunowa Pediatric sickle cell disease treatment, particularly the use of hydroxyurea, NSAIDs, and opioids, has evolved in response to clinical guidelines and regulatory shifts, but gaps still remain in how children receive essential medications. Joining Dr. Charles Turck to discuss these national prescribing trends, disparities in care, and the implications of evolving treatment guidelines for this population is Dr. Abiodun Ologunowa. Dr. Ologunowa is a doctoral candidate and research assistant in the Department of Pharmacy Practice and Clinical Research at the University of Rhode Island College of Pharmacy.

Guest: Abiodun Ologunowa Pediatric sickle cell disease treatment, particularly the use of hydroxyurea, NSAIDs, and opioids, has evolved in response to clinical guidelines and regulatory shifts, but gaps still remain in how children receive essential medications. Joining Dr. Charles Turck to discuss these national prescribing trends, disparities in care, and the implications of evolving treatment guidelines for this population is Dr. Abiodun Ologunowa. Dr. Ologunowa is a doctoral candidate and research assistant in the Department of Pharmacy Practice and Clinical Research at the University of Rhode Island College of Pharmacy.

Guest: Abiodun Ologunowa Pediatric sickle cell disease treatment, particularly the use of hydroxyurea, NSAIDs, and opioids, has evolved in response to clinical guidelines and regulatory shifts, but gaps still remain in how children receive essential medications. Joining Dr. Charles Turck to discuss these national prescribing trends, disparities in care, and the implications of evolving treatment guidelines for this population is Dr. Abiodun Ologunowa. Dr. Ologunowa is a doctoral candidate and research assistant in the Department of Pharmacy Practice and Clinical Research at the University of Rhode Island College of Pharmacy.

Guest: Yogindra Persuad Despite the promise of curative therapies for sickle cell disease, access and cost barriers highlight the ongoing need for disease-modifying treatments like hydroxyurea. Tune in to hear Dr. Yogindra Persuad, a physician in the Department of Hematology at St. Jude Children's Research Hospital in Memphis, Tennessee, discuss a multimodal approach to care, emphasizing research on oral agents and broader symptom management beyond pain.

Guest: Yogindra Persuad Despite the promise of curative therapies for sickle cell disease, access and cost barriers highlight the ongoing need for disease-modifying treatments like hydroxyurea. Tune in to hear Dr. Yogindra Persuad, a physician in the Department of Hematology at St. Jude Children's Research Hospital in Memphis, Tennessee, discuss a multimodal approach to care, emphasizing research on oral agents and broader symptom management beyond pain.

Guest: Parul Rai Cardiopulmonary complications are a leading cause of early mortality in adults with sickle cell disease, with evidence showing that cardiac injury may begin as early as childhood. However, despite the severity of these issues, there are currently no uniform guidelines for asymptomatic cardiac screening in this population. Join Dr. Parul Rai, a physician in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee, to learn about current research, early detection strategies, and the need for more sensitive diagnostic markers to prevent severe cardiac outcomes in patients with sickle cell disease.

Guest: Yogindra Persuad For decades, hydroxyurea has been the cornerstone therapy for sickle cell disease, but new disease-modifying therapies, curative strategies, and gene therapies are expanding options for patients. While these advancing approaches come with some risks, they can help offer symptom relief, reduce complications, and give patients more choices when it comes to managing their sickle cell disease. Hear Dr. Yogindra Persuad, a physician in the Department of Hematology at St. Jude Children's Research Hospital in Memphis, Tennessee, walk through the history of sickle cell disease treatment and discuss the benefits and risks of these developing therapies.

Guest: Nidhi Bhatt Effective transition from pediatric to adult care should begin as early as age twelve for patients with sickle cell disease, incorporating factors like education, self-advocacy skills, and support from transition care coordinators. Tune in to hear Dr. Nidhi Bhatt, who works in the Department of Hematology at St. Jude Children's Hospital in Memphis, Tennessee, discuss how involving patients and families early on helps identify barriers and improve long-term outcomes.

Guest: Parul Rai Hydroxyurea and chronic transfusions remain cornerstone therapies in managing sickle cell disease, with proven benefits in reducing stroke risk and improving anemia. However, considerations remain, including their impact on cardiac health. Learn about these long-standing therapies and best practices for using them to manage sickle cell disease with Dr. Parul Rai. Dr. Rai is a physician in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee.

Guest: Parul Rai Hydroxyurea and chronic transfusions remain cornerstone therapies in managing sickle cell disease, with proven benefits in reducing stroke risk and improving anemia. However, considerations remain, including their impact on cardiac health. Learn about these long-standing therapies and best practices for using them to manage sickle cell disease with Dr. Parul Rai. Dr. Rai is a physician in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee.

Guest: Yogindra Persuad For decades, hydroxyurea has been the cornerstone therapy for sickle cell disease, but new disease-modifying therapies, curative strategies, and gene therapies are expanding options for patients. While these advancing approaches come with some risks, they can help offer symptom relief, reduce complications, and give patients more choices when it comes to managing their sickle cell disease. Hear Dr. Yogindra Persuad, a physician in the Department of Hematology at St. Jude Children's Research Hospital in Memphis, Tennessee, walk through the history of sickle cell disease treatment and discuss the benefits and risks of these developing therapies.

Guest: Nidhi Bhatt Effective transition from pediatric to adult care should begin as early as age twelve for patients with sickle cell disease, incorporating factors like education, self-advocacy skills, and support from transition care coordinators. Tune in to hear Dr. Nidhi Bhatt, who works in the Department of Hematology at St. Jude Children's Hospital in Memphis, Tennessee, discuss how involving patients and families early on helps identify barriers and improve long-term outcomes.

Guest: Parul Rai Cardiopulmonary complications are a leading cause of early mortality in adults with sickle cell disease, with evidence showing that cardiac injury may begin as early as childhood. However, despite the severity of these issues, there are currently no uniform guidelines for asymptomatic cardiac screening in this population. Join Dr. Parul Rai, a physician in the Department of Hematology at St. Jude's Hospital in Memphis, Tennessee, to learn about current research, early detection strategies, and the need for more sensitive diagnostic markers to prevent severe cardiac outcomes in patients with sickle cell disease.

CME credits: 1.00 Valid until: 14-07-2026 Claim your CME credit at https://reachmd.com/programs/cme/treatment-of-a-broader-population-of-patients-with-dmd/33167/ By completing this activity, learners will gain the skills and confidence to determine therapeutic candidacy and conduct appropriate assessments for patients with Duchenne Muscular Dystrophy (DMD) across the age spectrum, including older and non-ambulatory individuals. They will improve their ability to interpret and apply the latest clinical data to real-world scenarios and make informed decisions about treatment and monitoring. Through case-based education, learners will enhance their competence in aligning patients to appropriate therapies and managing long-term care in a rapidly evolving treatment landscape.=

CME credits: 1.00 Valid until: 14-07-2026 Claim your CME credit at https://reachmd.com/programs/cme/treatment-of-a-broader-population-of-patients-with-dmd/33167/ By completing this activity, learners will gain the skills and confidence to determine therapeutic candidacy and conduct appropriate assessments for patients with Duchenne Muscular Dystrophy (DMD) across the age spectrum, including older and non-ambulatory individuals. They will improve their ability to interpret and apply the latest clinical data to real-world scenarios and make informed decisions about treatment and monitoring. Through case-based education, learners will enhance their competence in aligning patients to appropriate therapies and managing long-term care in a rapidly evolving treatment landscape.=

CME credits: 1.00 Valid until: 09-05-2026 Claim your CME credit at https://reachmd.com/programs/cme/new-frontiers-in-the-treatment-of-dmd-across-the-age-spectrum/32740/ Explore the latest treatment options, age-specific management strategies, and clinical trial data for patients with Duchenne muscular dystrophy (DMD). Through expert-led discussions and real-world patient cases, you'll gain practical insights to help you confidently navigate today's ever-evolving treatment landscape and deliver personalized, informed care.=

CME credits: 1.00 Valid until: 09-05-2026 Claim your CME credit at https://reachmd.com/programs/cme/new-frontiers-in-the-treatment-of-dmd-across-the-age-spectrum/32740/ Explore the latest treatment options, age-specific management strategies, and clinical trial data for patients with Duchenne muscular dystrophy (DMD). Through expert-led discussions and real-world patient cases, you'll gain practical insights to help you confidently navigate today's ever-evolving treatment landscape and deliver personalized, informed care.=

Guest: Jordan Orange, MD, PhD Guest: Joshua Milner, MD Guest: Eric Silver, MD Guest: Steven Lobritto, MD On this episode of Advances in Care, host Erin Welsh explores the story behind the GUARDIAN study, where thousands of newborn babies have been screened against rare disease by sequencing their genes, and looking for more conditions than any of the current standard screening panels. First, she hears from Dr. Jordan Orange, Physician-in-Chief at Morgan Stanley Children's Hospital at NewYork-Presbyterian and Columbia, about why genetic testing is a promising way of not only catching treatable rare diseases in infants, but also expanding health equity and medical resources to marginalized populations. Erin also hears from Dr. Josh Milner, a pediatric immunologist who treated a patient with a rare form of SCID, or severe combined immune deficiency, also known as bubble boy disease that was detected in the GUARDIAN screening panel. SCID is a disease that typically occurs in 1 of 50,000 babies. But GUARDIAN caught two cases within the first 10,000 babies involved in the program, indicating that the rate of the disease might be higher than expected, and that the most accurate way to detect is through genetic screening. Dr. Steven Lobritto, a pediatric gastroenterologist, also weighs in on how genetic screening can help identify Wilson's disease, a copper storage …

Host: Jennifer Caudle, DO Guest: Naval Daver, MD Not only is CD123 one of the most common antigens expressed on the surface of acute myeloid leukemia (AML) tumors, but it's also associated with more proliferative disease that's resistant to standard therapies. Given its prevalence and potential implications, a number of different CD123-targeting approaches are under investigation, including antibody-drug conjugates, bi-specific antibodies, fusion protein, and CAR T-cell therapy. In light of these new approaches, Dr. Jennifer Caudle and Dr. Naval Daver discuss the importance of targeting CD123 in AML. Dr. Daver is a Professor and Director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center in Houston.