Paul Melmeyer, MPP, executive vice president, public policy and advocacy, Muscular Dystrophy Association, explains how adding Duchenne muscular dystrophy to the Recommended Unified Screening Panel reshapes early diagnosis, treatment timing, and family counseling.
The research portfolio director at the Muscular Dystrophy Association spoke about ongoing advances in ALS research and how they may inform approaches to therapeutic development. [WATCH TIME: 5 minutes]
Angela Lek, PhD, chief research officer at the Muscular Dystrophy Association, provided an outlook on the 2026 MDA meeting and what clinicians should look forward to in preparation.
The chief research officer of the Muscular Dystrophy Association outlined MDA’s growing focus on muscle regeneration research and explains why the 2026 MDA Conference is a must-attend meeting for clinicians. [WATCH TIME: 3 minutes]
The chief research officer of the Muscular Dystrophy Association outlined emerging scientific priorities and community-driven initiatives shaping the 2026 MDA Clinical and Scientific Conference. [WATCH TIME: 4 minutes]
Angela Lek, PhD, chief research officer of the Muscular Dystrophy Association, discusses the FDA approval of intrathecal onasemnogene abeparvovec (Itvisma; Novartis) and what it means for SMA treatment across older pediatric and adult populations.
Sharon Hesterlee, PhD, president and CEO of the Muscular Dystrophy Association, discussed the 2026 MDA Conference, the significance of its keynote speaker, and the evolving therapeutic landscape in neuromuscular medicine.
The executive vice president of public policy and advocacy at the Muscular Dystrophy Association discussed how collaboration can improve policy, funding, and care for patients with neuromuscular diseases. [WATCH TIME: 7 minutes]
UCB's Kygevvi becomes the first FDA-approved treatment for thymidine kinase 2 deficiency, offering hope to patients with this rare neuromuscular disorder.
New guidelines enhance gene therapy delivery for Duchenne muscular dystrophy, emphasizing multidisciplinary care and safety monitoring for optimal patient outcomes.
The chief medical adviser at the Muscular Dystrophy Association spoke about the specific safety considerations clinicians should take when prescribing and administering gene therapy for patients with Duchenne muscular dystrophy. [WATCH TIME: 3 minutes]
Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Barry Byrne, MD, PhD. [LISTEN TIME: 15 minutes]
The research portfolio director at the Muscular Dystrophy Association discussed the evolving ALS therapeutic landscape, highlighting advances in genetic and RNA-based interventions. [WATCH TIME: 6 minutes]
The research portfolio director at the Muscular Dystrophy Association highlighted advances in gene-targeted therapies and biomarker development as potential key drivers of progress in ALS.
The research portfolio director at the Muscular Dystrophy Association talked about recent advances in ALS research highlight the promise of gene-targeted treatments and the increasing utility of biomarkers for diagnosis. [WATCH TIME: 3 minutes]
The MyacarinG study post-hoc analysis demonstrated rozanolixizumab's potential to significantly reduce ocular symptoms in gMG, with consistent improvements across multiple scoring systems.
The pediatric neurologist at Children’s Hospital of Philadelphia gave perspective on some of the main issues the clinical community is figuring out with gene therapies and their integration to clinical practice. [WATCH TIME: 3 minutes]
The pediatric neurologist provided a clinical overview of the advances and roadblocks of gene therapy in neuromuscular disorders, which will be highlighted at the 2025 MDA Clinical & Scientific Conference.
The associate professor of neurology at Columbia University provided commentary on the rapid advancements in translating genetic research into ALS treatments and the challenges that remain. [WATCH TIME: 5 minutes]
The pediatric neurologist at Children’s Hospital of Philadelphia provided background on a notable track session at the upcoming MDA Clinical & Scientific Conference covering updates in gene therapy for neuromuscular conditions. [WATCH TIME: 4 minutes]
The associate professor of neurology at Columbia University gave clinical insight on what neurologists and other neuromuscular specialists can expect from the ALS/frontotemporal dementia track at this year’s MDA Clinical & Scientific Conference. [WATCH TIME: 3 minutes]
Matthew B. Harms, MD, an associate professor of neurology at Columbia University Irving Medical Center, an MDA Care Center, gave clinical insights on the advances in ALS genetic research and what clinicians can expect from the ALS/FTD track at the 2025 MDA Clinical & Scientific Conference.
The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota talked about the potential of investigating inducted pluripotent stem cells in neuromuscular diseases at MDA’s 2025 conference. [WATCH TIME: 8 minutes]
The clinical researcher at St. Jude Children's Research Hospital discussed the evolving treatment landscape for Charcot-Marie-Tooth disease, which will be featured in a session at the 2025 MDA Conference. [WATCH TIME: 7 minutes]
The chief medical advisor at the Muscular Dystrophy Association shared key highlights of what attendees can expect at the 2025 MDA Clinical and Scientific Conference. [WATCH TIME: 4 minutes]
The program assembles a collaborative team of in-house and external experts in research, clinical care, and regulatory affairs to navigate gene therapy programs through the drug development process.
Donald S. Wood, PhD, president and chief executive officer at the MDA, provided thoughts and perspectives on the promising design and educational attainment for the 2025 MDA Clinical & Scientific conference.
Prior to the 2025 MDA Conference, the vice president of Public Policy & Advocacy at the Muscular Dystrophy Association talked about the increasing interest in gene therapy in the field of neuromuscular diseases.
As part of our monthly clinician spotlight, NeurologyLive® highlighted neuromuscular-associated cardiomyopathy expert Forum Kamdar, MD, PhD, FACC, FHFSA, an advanced heart failure physician-scientist at the University of Minnesota.
The chief research officer of the Muscular Dystrophy Association talked about what is to be expected at the 2025 MDA Conference, held March 16-19, in Dallas, Texas. [WATCH TIME: 5 minutes]
