Podcasts about rare and orphan diseases

Guest: John A. Morren, MD, FAAN, FAANEM The symptom variability, prolonged asymptomatic periods, and inconsistent presentations of generalized myasthenia gravis (gMG) often lead to significant diagnostic delays and misdiagnoses. Based on recent studies, these delays then correlate with increased disease severity, longer time to symptom control, and worsened quality of life. Here to help uncover the hidden toll of diagnostic delays on patients with gMG is Dr. John Morren. He's the Program Director of the Neuromuscular Medicine Fellowship at Cleveland Clinic and an Associate Professor of Neurology at Cleveland Clinic Lerner College of Medicine of Case Western Reserve University.

Host: Gerard A. Silvestri MD, MS, Master FCCP Guest: Anurag Singh, MD Guest: Adam H. Fox, MD, MSc Guest: Mariam Alexander, MD, PhD Despite the typically poor prognosis of extensive-stage small cell lung cancer, recent advancements are reshaping clinical perspectives on treatment. New and emerging options show promise for prolonged survival and improved quality of life. Join Drs. Gerard Silvestri, Adam Fox, Mariam Alexander, and Anurag Singh as they discuss how the therapeutic landscape is evolving for these patients. Silvestri is a pulmonologist and the Hillenbrand Professor of Thoracic Oncology at the Medical University of South Carolina. Dr. Fox is a pulmonologist and Assistant Professor of Medicine at the Medical University of South Carolina. Dr. Alexander is a medical oncologist and Assistant Professor of Medicine at the Medical University of South Carolina. Dr. Singh is a Professor of Radiation Oncology and the Director of Radiation Research at the Roswell Park Cancer Center in New York. This program is produced in partnership with the American College of Chest Physicians and is sponsored by AstraZeneca.

Host: Gerard A. Silvestri MD, MS, Master FCCP Guest: Anurag Singh, MD Guest: Adam H. Fox, MD, MSc Guest: Mariam Alexander, MD, PhD Recent therapeutic advances are reshaping our approach to limited-stage small cell lung cancer. In this multidisciplinary discussion, Dr. Gerard Silvestri sits down with Drs. Adam Fox, Mariam Alexander, and Anurag Singh to explore the evolving standard of care and practical considerations for timely and effective care. Dr. Silvestri is a pulmonologist and the Hillenbrand Professor of Thoracic Oncology at the Medical University of South Carolina. Dr. Fox is a pulmonologist and Assistant Professor of Medicine at the Medical University of South Carolina. Dr. Alexander is a medical oncologist and Assistant Professor of Medicine at the Medical University of South Carolina. Dr. Singh is a Professor of Radiation Oncology and the Director of Radiation Research at the Roswell Park Cancer Center in New York. This program is produced in partnership with the American College of Chest Physicians and is sponsored by AstraZeneca.

Guest: Divya Shankar, MD A recent study investigated whether pulse-dose corticosteroids offer a benefit over lower doses in managing acute exacerbations of idiopathic pulmonary fibrosis (IPF). Join Dr. Divya Shankar as she explains the real-world data, variability in prescribing patterns, and outcomes observed in different levels of care. Divya Shankar is an Assistant Professor of Medicine at Boston University Chobanian and Avedisian School of Medicine as well as a Pulmonary and Critical Care Physician at Boston Medical Center, and she spoke about this topic at the 2025 American Thoracic Society International Conference.

Guest: Rosaline Quinlivan As patients with Duchenne muscular dystrophy (DMD) transition from pediatric to adult care, they may encounter healthcare professionals—from primary care to emergency medicine—without prior experience managing their condition. Explore critical gaps in adult services for DMD and gain practical insights to help deliver timely, informed, and compassionate care with Dr. Rosaline Quinlivan, Professor of Neuromuscular Disease at University College London.

Guest: Rosaline Quinlivan From sustained therapy and emergency planning to psychological support, coordinated care is essential as patients with Duchenne muscular dystrophy (DMD) transition into adulthood. Explore multidisciplinary approaches to optimize function, independence, and quality of life in adults with DMD with Dr. Rosaline Quinlivan, Professor of Neuromuscular Disease at University College London.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Jason Ackrivo, MD, MSCE Guest: Bethany Lussier, MD Respiratory decline in patients with neuromuscular diseases and mitochondrial myopathies can be challenging to identify, especially because its early signs may be subtle and vary from person to person. However, knowing how to evaluate a patient's respiratory function is key to improving outcomes and quality of life. Joining Dr. Charles Turck to discuss this importance and provide recommendations for optimizing respiratory care are Drs. Jason Ackrivo and Bethany Lussier. Dr. Ackrivo is an Assistant Professor of Medicine in Pulmonary, Allergy, and Critical Care at the Hospital of the University of Pennsylvania, and Dr. Lussier is an Associate Professor of Internal Medicine at UT Southwestern Medical Center and a member of its Division of Pulmonary and Critical Care Medicine.

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: Charles Turck, PharmD, BCPS, BCCCP Guest: Aaron Logan, MD, PhD Although acute lymphoblastic leukemia (ALL) is a relatively rare disease with fewer than 7,000 people diagnosed each year in the US,1,2 there's been a lot of progress in the management of B-cell ALL over the years. One key development comes from the E1910 Phase III trial, which explored the addition of blinatumomab to frontline consolidation chemotherapy for patients with Philadelphia chromosome-negative B-lineage ALL (B-ALL).3 Joining Dr. Charles Turck to discuss the impacts of this data on B-ALL clinical guidelines and practice is Dr. Aaron Logan. Dr. Logan is a Professor of Clinical Medicine in the Division of Hematology/Oncology and Director of the Hematologic Malignancies Tissue Bank at UCSF. References: National Cancer Institute. SEER Cancer Stat Facts: Acute Lymphocytic Leukemia (ALL). Accessed at https://seer.cancer.gov/statfacts/html/alyl.html on March 31, 2025 Dana-Farber Cancer Institute. Acute Lymphoblastic Leukemia (ALL). Accessed September 17, 2024. https://www.dana-farber.org/cancer-care/types/acute-lymphoblastic-leukemia Litzow MR, et al. Blood. 2022;140(suppl 2):LBA-1

Guest: Jennie Taylor, MD The FDA approval of vorasidenib marks a new era for mutant isocitrate dehydrogenase (mIDH) gliomas. Approved in 2024 for grade 2 tumors after surgery, it doubled progression-free survival in the INDIGO trial. Dr. Jennie Taylor, Associate Professor of Neurology and Neurological Surgery at the University of California San Francisco, dives into the data and explains what questions remain about long-term use and broader applications.

Guest: Jennie Taylor, MD Defined by the production of 2-hydroxyglutarate, mutant isocitrate dehydrogenase (mIDH) gliomas are diffuse, slow-growing tumors. Managing these tumors requires personalized strategies that consider resectability, histology, and long-term treatment impacts. Dr. Jennie Taylor, Assistant Professor of Neurology and Neurological Surgery at the University of California San Francisco, explains the complexities behind this type of tumor and implications for patient care. Dr. Taylor also spoke about this topic at the 2025 American Academy of Neurology Annual Meeting.

Guest: Lauren Schaff, MD Glioblastomas are fast, aggressive, and resistant to many standard therapies. Dr. Lauren Schaff, a neuro-oncologist at Memorial Sloan Kettering Cancer Center, explains how new molecular understandings and treatment avenues are paving the way for a more personalized, hopeful approach to care.

Guest: Jennie Taylor, MD Defined by the production of 2-hydroxyglutarate, mutant isocitrate dehydrogenase (mIDH) gliomas are diffuse, slow-growing tumors. Managing these tumors requires personalized strategies that consider resectability, histology, and long-term treatment impacts. Dr. Jennie Taylor, Assistant Professor of Neurology and Neurological Surgery at the University of California San Francisco, explains the complexities behind this type of tumor and implications for patient care. Dr. Taylor also spoke about this topic at the 2025 American Academy of Neurology Annual Meeting.

Guest: Lauren Schaff, MD Glioblastomas are fast, aggressive, and resistant to many standard therapies. Dr. Lauren Schaff, a neuro-oncologist at Memorial Sloan Kettering Cancer Center, explains how new molecular understandings and treatment avenues are paving the way for a more personalized, hopeful approach to care.

Guest: Jennie Taylor, MD Defined by the production of 2-hydroxyglutarate, mutant isocitrate dehydrogenase (mIDH) gliomas are diffuse, slow-growing tumors. Managing these tumors requires personalized strategies that consider resectability, histology, and long-term treatment impacts. Dr. Jennie Taylor, Assistant Professor of Neurology and Neurological Surgery at the University of California San Francisco, explains the complexities behind this type of tumor and implications for patient care. Dr. Taylor also spoke about this topic at the 2025 American Academy of Neurology Annual Meeting.

Guest: Lauren Schaff, MD Glioblastomas are fast, aggressive, and resistant to many standard therapies. Dr. Lauren Schaff, a neuro-oncologist at Memorial Sloan Kettering Cancer Center, explains how new molecular understandings and treatment avenues are paving the way for a more personalized, hopeful approach to care.

Guest: Jennie Taylor, MD The FDA approval of vorasidenib marks a new era for mutant isocitrate dehydrogenase (mIDH) gliomas. Approved in 2024 for grade 2 tumors after surgery, it doubled progression-free survival in the INDIGO trial. Dr. Jennie Taylor, Associate Professor of Neurology and Neurological Surgery at the University of California San Francisco, dives into the data and explains what questions remain about long-term use and broader applications.

Guest: Jennie Taylor, MD The FDA approval of vorasidenib marks a new era for mutant isocitrate dehydrogenase (mIDH) gliomas. Approved in 2024 for grade 2 tumors after surgery, it doubled progression-free survival in the INDIGO trial. Dr. Jennie Taylor, Associate Professor of Neurology and Neurological Surgery at the University of California San Francisco, dives into the data and explains what questions remain about long-term use and broader applications.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Kaitlin Batley, MD Guest: Esra Caylan, MD Thymidine kinase 2 deficiency (TK2d) presents with a variability of symptoms, including respiratory, neurological, and ocular ones, making it difficult to diagnose. Because of this, a multidisciplinary care team that's tailored to each patient's needs is key to effectively identifying and treating this disease. Hear about the role of a multidisciplinary team and strategies for personalizing treatment with Drs. Kaitlin Batley and Esra Caylan. Dr. Batley is the Director of Pediatric Neuromuscular Medicine at Children's Health and an Assistant Professor at UT Southwestern Medical Center. Dr. Caylan is a pediatric pulmonologist at Children's Health and an Assistant Professor in the Department of Pediatrics at UT Southwestern Medical Center.

Guest: Hans Katzberg, MD Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a complex immune-mediated condition affecting the peripheral nervous system. Join Dr. Hans Katzberg, Professor of Medicine at the University of Toronto, as he explains the pathophysiology behind CIDP, risk factors in disease development, and diagnostic strategies.

Guest: Sami Khella, MD With personalized approaches, alternatives to steroids, and novel therapies like efgartigimod, the treatment landscape for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is continuing to evolve. Join Dr. Sami Khella as he shares expert insights on our available options. Dr. Khella is the Director of Clinical Electrophysiology and a Professor of Clinical Neurology at the University of Pennsylvania, and he spoke about this topic at the 2025 American Academy of Neurology Annual Meeting.

Guest: Sami Khella, MD With personalized approaches, alternatives to steroids, and novel therapies like efgartigimod, the treatment landscape for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is continuing to evolve. Join Dr. Sami Khella as he shares expert insights on our available options. Dr. Khella is the Director of Clinical Electrophysiology and a Professor of Clinical Neurology at the University of Pennsylvania, and he spoke about this topic at the 2025 American Academy of Neurology Annual Meeting.

Guest: Sami Khella, MD Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) can present in multiple ways, making diagnosis complex. Dr. Sami Khella joins the program to outline how we can distinguish CIDP from similar neurological disorders and explain how response to therapy can guide diagnosis. Dr. Khella is the Director of Clinical Electrophysiology and a Professor of Clinical Neurology at the University of Pennsylvania, and he spoke about this topic at the 2025 American Academy of Neurology Annual Meeting.

Host: Nathan Falk, MD, MBA, FAAFP Guest: Ashwin Basavaraj, MD Early recognition and diagnosis of non-tuberculosis mycobacteria (NTM) and bronchiectasis are key for optimal patient management. However, these conditions are often misdiagnosed as COPD or asthma, leading to significant challenges and delays in treatment. Join Drs. Nate Falk and Ashwin Basavaraj as they share perspectives on accurately diagnosing NTM and bronchiectasis with early symptom recognition, a thorough patient history, and testing. Dr. Falk is a board-certified family medicine physician, a Professor and Founding Residency Director for Family Medicine at Florida State University in partnership with BayCare Health System, and the Assistant Dean for Graduate Medical Education at Florida State University. Dr. Basavaraj is an Associate Professor of Medicine at New York University Grossman School of Medicine, the Director of the Bronchiectasis and NTM Education Program at NYU Langone Health, and the Section Chief of Pulmonary, Critical Care, and Sleep Medicine at Bellevue Hospital Center. This program is produced in partnership with the American College of CHEST Physicians and is sponsored by Insmed Incorporated.

Host: Michelle Kittleson, MD, PhD Transthyretin cardiac amyloidosis (ATTR-CM) is an underrecognized disorder, leading to a variety of unmet needs for patients. Dr. Michelle Kittleson, Director of Postgraduate Education in Heart Failure and Transplantation and Professor of Medicine at the Cedars-Smidt Heart Institute, explores these challenges and discusses how early recognition, genetic screening, and emerging therapies can help improve patient outcomes.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Anthony Stein, MD Could a novel triplet combination therapy offer a new path forward in acute myeloid leukemia (AML) treatment? A recent Phase 1B trial explored the potential of tagraxofusp, azacitidine, and venetoclax for AML patients. To discuss the findings on this triplet combination therapy, Dr. Charles Turck speaks with Dr. Anthony Stein, Professor in the Department of Hematology and Hematopoietic Cell Transplantation at City of Hope in Duarte, California.

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.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Yi Bin Chen, MD Guest: Olaf Penack, MD For the past several decades, standard treatment for acute graft-versus-host disease (aGVHD) has been systemic high-dose steroids.1 While essential in some ways to initially control aGVHD, steroids are not beneficial in the long run due to the immunosuppression and toxicities associated with high cumulative doses.1,2 So what can be done moving forward to improve first-line steroid response and minimize cumulative steroid exposure? Joining Dr Charles Turck to discuss the potential of nonimmunosuppressive steroid-sparing agents for the first-line treatment of aGVHD are Drs Yi Bin Chen and Olaf Penack. Dr Chen is the Director of the Hematopoietic Cell Transplant and Cell Therapy Program at Massachusetts General Hospital, and Dr Penack is a senior physician and principal investigator within Hematology and Oncology at Charité Berlin. References: Bell EJ, Yu J, Bhatt V, et al. Healthcare resource utilization and costs of steroid-associated complications in patients with graft-versus-host disease. Transplant and Cell Ther. 2022;28(10):707.e1-707.e7. Martin PJ, Rizzo JD, Wingard JR, et al. First- and second-line systemic treatment of acute graft-versus-host disease: recommendations of the American Society of Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2012;18(8):1150-1163. © 2025 CSL BehringCMD-964-0016-JAN25

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Bimaje Akpa, MD Clinical practice guidelines from the American College of Chest Physicians provide recommendations for managing respiratory complications in patients with neuromuscular diseases, which can help inform our evaluation and treatment decisions. Joining Dr. Charles Turck to break down key insights from the CHEST guidelines and their impacts on clinical practice is Dr. Bimaje Akpa. Dr. Akpa is an Assistant Professor of Medicine in the Division of Pulmonary, Allergy, Critical Care, and Sleep Medicine at the University of Minnesota.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Jason Ackrivo, MD, MSCE Respiratory complications are fairly common in patients with neuromuscular disorders, and given their vast impacts on a patient's prognosis and quality of life, it's critical to proactively detect and monitor any signs of respiratory decline. To learn more about the symptoms, impacts, and diagnosis of respiratory complications in patients with neuromuscular disorders, Dr. Charles Turck speaks with Dr. Jason Ackrivo. Not only is Dr. Ackrivo the Associate Director of the Fishman Program for Home Assisted Ventilation, but he's also an Assistant Professor of Medicine in Pulmonary, Allergy, and Critical Care at the Hospital of the University of Pennsylvania.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Hank Mayer, MD Patients with neuromuscular conditions often exhibit respiratory symptoms that impact their airway clearance and gas exchange, making it increasingly difficult for them to breathe. However, effective assessment techniques and early intervention can help mitigate the progression of this dysfunction, potentially keeping patients from needing extended recovery time or even inpatient care. Joining Dr. Charles Turck to discuss the importance of informed, proactive intervention for respiratory symptoms in patients with neuromuscular conditions is Dr. Hank Mayer. Dr. Mayer is the Medical Director of the Pulmonary Function Laboratory at the Children's Hospital of Philadelphia and a Professor of Clinical Pediatrics at the University of Pennsylvania's Perelman School of Medicine.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Miguel-Angel Perales, MD Guest: Anna Sureda, MD, PhD Unfortunately, the treatment paradigm for acute graft-versus-host disease (aGVHD) has not changed for a couple of decades as steroids remain the first-line treatment.1 However, steroids are not a targeted therapy, and they have many side effects that are not well tolerated by patients, including life-threatening infections.1-3 Given these risks, it is important to know how to manage infections related to immunosuppression and optimize the care of patients with aGVHD. Joining Dr Charles Turck to discuss the current landscape of aGVHD management are Dr Miguel-Angel Perales and Dr Anna Sureda. Dr Miguel-Angel Perales is the Chief of the Adult Bone Marrow Transplantation Service at Memorial Sloan Kettering Cancer Center in New York, and Dr Sureda is the Head of Clinical Hematology at the University of Barcelona in Spain. References: Bell EJ, Yu J, Bhatt V, et al. Healthcare resource utilization and costs of steroid-associated complications in patients with graft-versus-host disease. Transplant Cell Ther. 2022;28(10):707.e1-707.e7. doi:10.1016/j.jtct.2022.04.014 Sullivan PW, Ghushchyan VH, Globe G, Sucher B. Health-related quality of life associated with systemic steroids. Qual Life Res. 2017;26(4):1037-1058. García-Cadenas I, Rivera I, Martino R, et al. Patterns of infection and infection-related mortality in patients with steroid-refractory acute graft versus host disease. Bone Marrow …

Guest: Gil I. Wolfe MD, FAAN Rozanolixizumab is a fast-acting myasthenia gravis treatment that reduces IgG levels by blocking the neonatal Fc receptor. Dr. Gil Wolfe explains how it compares to other treatment options like corticosteroids, taking side effects and quality of life into consideration. Dr. Wolf is a SUNY Distinguished Professor of Neurology at the University of Buffalo Jacobs School of Medicine and Biomedical Sciences.

CME credits: 1.00 Valid until: 31-12-2025 Claim your CME credit at https://reachmd.com/programs/cme/recent-guidelines-updates-implications-to-practice/26659/ This series of bite-sized episodes will take you on the winding journey that patients with idiopathic multicentric Castleman disease (iMCD) often undergo to achieve an accurate diagnosis. Once the condition is finally identified, even the most astute specialists can find themselves challenged by the treatment, management, and monitoring of these patients. Join Drs. Corey Casper and Sudipto Mukherjee as they explain the ups and downs of this rare disease.

Guest: Mateo Bustamante Idiopathic multicentric Castleman disease (iMCD) is a rare, life-threatening condition. Early diagnosis through excisional biopsies is key for speeding up treatment and improve patient outcomes. Learn more with Mateo Bustamante, a Senior Clinical Data Analyst at the Center for Cytokine Storm Treatment and Laboratory at University of Pennsylvania's Perelman School of Medicine.

Guest: Joshua Brandstadter, MD, PhD, MSc Castleman disease is difficult to diagnose and treat, with half of patients not responding to therapy. To address these challenges, Dr. Joshua Brandstadter discusses his research on how stromal cells play a role in the disease. Based at the University of Pennsylvania, Dr. Brandstadter is an Instructor of Medicine in the Division of Hematology and Oncology and the Director of Clinical Research for the Center of Cytokine Storm Treatment and Laboratory.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Mary Frances McMullin, MD The MOMENTUM study evaluated the impact of the treatment option momelotinib on patient-reported outcomes, including symptom burden and overall quality of life. Joining Dr. Charles Turck to share the key findings and potential implications for myelofibrosis care is Dr. Mary Francis McMullin, who co-authored and presented the poster at the American Society of Hematology (ASH) Annual Meeting and Exposition.

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Douglas Tremblay, MD Based on three randomized-controlled trials, the JAK inhibitor momelotinib has demonstrated clinical activity against anemia, constitutional symptoms, and splenomegaly in patients with myelofibrosis. But what do we know about its long-term safety? That's what a recent study explored, and now, Dr. Douglas Trembley is here to share the findings from the largest clinical trial safety database for a JAK inhibitor in myelofibrosis. Dr. Trembley is an Assistant Professor at the Icahn School of Medicine at Mount Sinai in New York.

Host: Gates B. Colbert, MD Guest: Ryan Cassaday, MD According to the available long-term data, CAR T-cell therapy boosts overall survival and durable remission rates in patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL). Given this data, it's important to know how we can best identify appropriate patients for this approach and manage adverse events so they can achieve those long-term benefits. To learn more about the available data and key considerations for using CAR T-cell therapy to treat relapsed/refractory B-ALL, Dr. Gates Colbert speaks with Dr. Ryan Cassaday, Associate Professor in the Division of Hematology-Oncology at the University of Washington School of Medicine in Seattle.

CME credits: 1.00 Valid until: 11-10-2025 Claim your CME credit at https://reachmd.com/programs/cme/advancing-cystic-fibrosis-addressing-disparities-challenging-perceptions-and-innovating-patient-centered-care/24487/ Cystic Fibrosis (CF) care faces challenges from health disparities, limited access, and unintended bias, affecting patient outcomes. This CME program aims to equip healthcare professionals with the skills needed to overcome these barriers and provide equitable care for all CF patients. It covers telehealth strategies, best practices for transitioning patients from pediatric to adult care, and managing co-morbidities. The program also explores how evolving treatments impact patient outcomes.=

Host: Matt Birnholz, MD The SIMPLIFY-2 trial compared the efficacy of momelotinib to best available therapy, which was primarily continued ruxolitinib, in patients with myelofibrosis and anemia who were previously treated with a JAK inhibitor. According to the findings, momelotinib potentially offers better outcomes and a more comprehensive management strategy by addressing the underlying molecular mechanism of anemia. Learn more about the trial design and results with Dr. Matt Birnholz.

CME credits: 1.00 Valid until: 25-09-2025 Claim your CME credit at https://reachmd.com/programs/cme/mastering-the-complexity-of-aml-treatment-a-multidimensional-approach-to-diagnosis-therapy-and-side-effect-management/26723/ While the expanding arsenal of therapies available for patients with AML allows practitioners to further hone treatment and individualize care, the complexity of treatment decision-making requires careful consideration of potential risks and benefits of each treatment option. This real-world, case-based program will provide a format for community hematology-oncologists to hear current updates on the latest targeted therapies in AML and engage in pointed, practical discussions on how to best integrate these data into current and local AML treatment paradigms.=

Guest: Betsy O'Donnell, MD Plasma cell disorders range from multiple myeloma to the monoclonal gammopathy of undetermined significance (MGUS), which is a benign condition that affects anywhere between 3 and 10 percent of the population starting at age 50. However, about 1 percent of people per year who have MGUS will progress to multiple myeloma. Here to talk about common precursor diseases like MGUS and smoldering myeloma and how they can progress to multiple myeloma is Dr. Elizabeth O'Donnell, Director of Early Detection and Prevention at Dana-Farber.

Host: Jennifer Caudle, DO Guest: Samuel R. Wilson, MD Sickle cell disease is among the most common inherited conditions globally, affecting more than seven million individuals worldwide.1-3 Given its prevalence, it is important to understand all of the complexities surrounding this disease. Joining Dr. Jennifer Caudle to discuss the pathophysiology, clinical presentation, burden, and unmet needs of sickle cell disease is Dr. Samuel R. Wilson, Assistant Professor of Medicine in the Division of Hematology at the University of North Carolina School of Medicine. References: Sedrak A, Kondamudi NP. Sickle cell disease. StatPearls Publishing; 2023. Updated August 12, 2023. Accessed April 16, 2024. https://www.ncbi.nlm.nih.gov/books/NBK482384/. Thomson AM, McHugh TA, Oron AP, et al. Global, regional, and national prevalence and mortality burden of sickle cell disease, 2000–2021: a systematic analysis from the Global Burden of Disease Study 2021. Lancet Haematol. 2023;10(8):e585-e599. doi:10.1016/S2352-3026(23)00118-7. CDC. Data & statistics on sickle cell disease. Centers for Disease Control and Prevention. Published May 2, 2022. Accessed April 16, 2024. https://www.cdc.gov/ncbddd/sicklecell/data.html/. Agios Pharmaceuticals, Inc. © 2024 All right reserved.SCD-US-0095 / June 2024

Host: Jennifer Caudle, DO Guest: Samuel R. Wilson, MD Sickle cell disease is among the most common inherited conditions globally, affecting more than seven million individuals worldwide.1-3 Given its prevalence, it is important to understand all of the complexities surrounding this disease. Joining Dr. Jennifer Caudle to discuss the pathophysiology, clinical presentation, burden, and unmet needs of sickle cell disease is Dr. Samuel R. Wilson, Assistant Professor of Medicine in the Division of Hematology at the University of North Carolina School of Medicine. References: Sedrak A, Kondamudi NP. Sickle cell disease. StatPearls Publishing; 2023. Updated August 12, 2023. Accessed April 16, 2024. https://www.ncbi.nlm.nih.gov/books/NBK482384/. Thomson AM, McHugh TA, Oron AP, et al. Global, regional, and national prevalence and mortality burden of sickle cell disease, 2000–2021: a systematic analysis from the Global Burden of Disease Study 2021. Lancet Haematol. 2023;10(8):e585-e599. doi:10.1016/S2352-3026(23)00118-7. CDC. Data & statistics on sickle cell disease. Centers for Disease Control and Prevention. Published May 2, 2022. Accessed April 16, 2024. https://www.cdc.gov/ncbddd/sicklecell/data.html/. Agios Pharmaceuticals, Inc. © 2024 All right reserved.SCD-US-0095 / June 2024

Host: Matt Birnholz, MD Guest: Saad Kenderian M.B, Ch.B CAR T-cell therapy has been revolutionary in the treatment of blood cancers like chronic lymphocytic leukemia and multiple myeloma, and according to recent research, this therapeutic approach may also help patients with thyroid cancer. However, there are several challenges associated with applying this technology to target solid tumors in thyroid cancer, like tumor heterogeneity and resistance. Joining Dr. Matt Birnholz to talk about these challenges and how a research team is working to overcome them is Dr. Saad J. Kenderian, Assistant Professor of Oncology, Immunology, and Medicine at the Mayo Clinic in Rochester, Minnesota.

CME credits: 1.00 Valid until: 04-06-2025 Claim your CME credit at https://reachmd.com/programs/cme/applexus-hot-topics-2024-clinical-updates-on-non-covalent-btk-inhibitors-in-cll-and-mcl/26980/ On-demand webcast of expert faculty presentation and case discussion on treating patients with CLL and MCL with non-covalent BTK inhibitors, as presented at CCO and PCE's APPlexus Hot Topics 2024.=

CME credits: 1.00 Valid until: 29-05-2025 Claim your CME credit at https://reachmd.com/programs/cme/whim-syndrome-treatment-we-can-do-better/24550/ WHIM syndrome is a rare, inherited, combined immunodeficiency disease caused by reduced mobilization and trafficking of white blood cells from the bone marrow due to over-signaling of the CXCR4/CXCL12 pathway. WHIM syndrome is named for its four common clinical findings. The diagnostic challenges arise because not all symptoms are required for a diagnosis and all of them do not appear at the same time. Patients have deficient blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), which results in frequent, recurrent infections. Affected individuals are particularly susceptible to human papillomavirus (HPV), which can cause skin and genital warts and potentially lead to certain types of cancer. Our program goal is to identify risk factors associated with WHIM syndrome, focusing on making an earlier diagnosis that can impact long-term outcomes, timely treatment options and ultimately improved quality of life for patients.

CME credits: 1.00 Valid until: 29-05-2025 Claim your CME credit at https://reachmd.com/programs/cme/whim-syndrome-and-cxcr4-variants-new-insights-into-cellular-changes-that-can-impact-patient-treatment-plans/24548/ WHIM syndrome is a rare, inherited, combined immunodeficiency disease caused by reduced mobilization and trafficking of white blood cells from the bone marrow due to over-signaling of the CXCR4/CXCL12 pathway. WHIM syndrome is named for its four common clinical findings. The diagnostic challenges arise because not all symptoms are required for a diagnosis and all of them do not appear at the same time. Patients have deficient blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), which results in frequent, recurrent infections. Affected individuals are particularly susceptible to human papillomavirus (HPV), which can cause skin and genital warts and potentially lead to certain types of cancer. Our program goal is to identify risk factors associated with WHIM syndrome, focusing on making an earlier diagnosis that can impact long-term outcomes, timely treatment options and ultimately improved quality of life for patients.

CME credits: 1.00 Valid until: 29-05-2025 Claim your CME credit at https://reachmd.com/programs/cme/you-dont-know-whim-syndrome-a-chronic-neutropenic-disorder/24547/ WHIM syndrome is a rare, inherited, combined immunodeficiency disease caused by reduced mobilization and trafficking of white blood cells from the bone marrow due to over-signaling of the CXCR4/CXCL12 pathway. WHIM syndrome is named for its four common clinical findings. The diagnostic challenges arise because not all symptoms are required for a diagnosis and all of them do not appear at the same time. Patients have deficient blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), which results in frequent, recurrent infections. Affected individuals are particularly susceptible to human papillomavirus (HPV), which can cause skin and genital warts and potentially lead to certain types of cancer. Our program goal is to identify risk factors associated with WHIM syndrome, focusing on making an earlier diagnosis that can impact long-term outcomes, timely treatment options and ultimately improved quality of life for patients.