Muscular Dystrophy Association

The assistant professor in the Department of Chemistry and Biochemistry at University of Nevada, Las Vegas reflected on receiving the first MDA Research Momentum Award at the 2026 MDA Conference. [WATCH TIME: 2 minutes]

The chief research officer of the Muscular Dystrophy Association spoke on the FDA's approval of a high-dose regimen of nusinersen and how it fits into an increasingly complex treatment landscape. [WATCH TIME: 11 minutes]

The founder and CEO at the Hereditary Neuropathy Foundation discussed how learning about patient experiences has influenced research in Charcot-Marie-Tooth disease. [WATCH TIME: 5 minutes]

The 2026 MDA National Ambassador discussed the importance of connecting patients’ experiences with scientific understanding, as well as fostering a sense of community among those living with neuromuscular disease. [WATCH TIME: 2 minutes]

The chief research officer of the Muscular Dystrophy Association spoke on highlights and themes from the organization's 2026 Clinical and Scientific Conference. [WATCH TIME: 9 minutes]

The research portfolio director at the Muscular Dystrophy Association highlighted the collaborative spirit, the expanding therapeutic pipeline, and the growing emphasis on patients’ voices in neuromuscular disease on display at MDA 2026. [WATCH TIME: 8 minutes]

Early BB-301 gene therapy data show durable swallowing gains and benign safety in OPMD dysphagia, hinting at disease modification.

Interim data from the phase 1/2 AFFINITY DUCHENNE trial suggest RGX-202 gene therapy was well tolerated and produced robust microdystrophin expression with early functional improvements in boys with Duchenne muscular dystrophy.

Interim results from the phase 1/2 FORTIS trial suggest the investigational gene therapy AT845 was generally well tolerated and associated with stable respiratory and functional outcomes in adults with late-onset Pompe disease.

The professor of neurology at Columbia University Irving Medical Center highlighted the decades of research collaboration for patients with neuromuscular disease at the 2026 MDA conference.

The phase 1/2 VALOR trial is evaluating the safety and preliminary efficacy of the investigational gene therapy ASP2957 in infants with X-linked myotubular myopathy who require significant ventilatory support.

Long-term follow-up from an open-label phase 2 extension study suggests the investigational exon-skipping therapy brogidirsen was well tolerated over 4.5 years and may help maintain motor function in patients with Duchenne muscular dystrophy.

Interim phase 2 data from the FORWARD-53 study suggest the exon-skipping therapy WVE-N531 was well tolerated and produced sustained dystrophin expression, reduced fibrosis, and functional improvements in boys with DMD amenable to exon 53 skipping.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, also spoke about what he personally is most looking forward to at this year's meeting.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Jeff Chamberlain, PhD. [LISTEN TIME: 23 minutes]

The chief medical advisor at the Muscular Dystrophy Association also discussed what he personally is looking forward to at the conference this year. [WATCH TIME: 4 minutes]

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.