The Urgency of Funding and Equity in Neuromuscular Research and Care: Robert Califf, MD
March 27th 2025The former FDA commissioner spoke on the need for policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities. [WATCH TIME: 4 minutes]
WVE-N531 Improves Muscle Health in Latest Phase 2 Trial of Duchenne Muscular Dystrophy
March 27th 2025WVE-N531, an investigational exon-skipping therapy, showed promising 48-week results in the FORWARD-53 study, marking the first-ever improvement in muscle health for patients with Duchenne muscular dystrophy, with plans for an NDA filing in 2026.
Raising Global Awareness for Epilepsy on Purple Day: Sonal Bhatia, MD, FACNS
March 26th 2025The director of the pediatric epilepsy monitoring unit at the Medical University of South Carolina provided commentary on the significance of Purple Day as a means to raise awareness about epilepsy. [WATCH TIME: 3 minutes]
Epilepsy Unveiled: Addressing Myths, Treatment Gaps, and New Innovations
March 26th 2025Luis Tornes, MD, a neurologist and director of Baptist Health’s Epilepsy Program, provided clinical insights on raising awareness for Purple Day, a global initiative dedicated for patients and families with epilepsy.
Differentiating Cognitive Decline in Multiple Sclerosis and Alzheimer Disease: Sarah Levy, PhD
March 25th 2025The assistant professor in the department of neurology at Mount Sinai discussed distinguishing cognitive impairment in MS from AD emphasizing orientation as a key differentiator. [WATCH TIME: 5 minutes]
Addressing Persistent Challenges in NMOSD Diagnosis and Treatment: Sumaira Ahmed
March 24th 2025Despite advancements in NMOSD treatments, the founder and executive director of the Sumaira Foundation discussed how misdiagnosis, delayed diagnosis, and disparities in care remain significant challenges. [WATCH TIME: 4 minutes]
MDA Study Highlights Lack of Psychosocial Care Services for Duchenne Muscular Dystrophy
March 23rd 2025Despite facing mental health concerns with anger, aggression, or irritability, among others, slightly less than one-fourth of patients with DMD utilized psychosocial services such as counseling or therapy.
Celebrating Milestones and Tackling New Frontiers in Pompe Disease: Priya Kishnani, MD
March 21st 2025The professor of pediatrics and division chief of Medical Genetics at Duke University gave clinical insight on a presentation on the advancements and limitations of enzyme replacement therapy for Pompe Disease. [WATCH TIME: 3 minutes]