The director of the Neuromuscular Division at the Medical University of South Carolina shared post hoc CHAMPION-MG trial findings at MDA 2026, suggesting ravulizumab may enable corticosteroid reduction while maintaining or improving clinical outcomes. [WATCH TIME: 4 minutes]
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]
Diana Castro, MD, founder and director of the Neurology and Neuromuscular Care Center, discussed early phase 1b data on salanersen, an investigational therapy for patients living with spinal muscular atrophy.
The director of neuromuscular medicine at Marcus Neuroscience Institute shared findings from an analysis presented at MDA 2026 that compared real-world infection rates associated with generalized myasthenia gravis therapies. [WATCH TIME: 8 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]
Nazem Atassi, MD, SVP and Global Development Head of Neurology & Gene Therapy at Novartis, discussed 64-week STEER data and the evolving role of one-time gene therapy in SMA care.
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 chief medical officer at Edgewise discussed long-term MESA data showing sustained functional stabilization with sevasemten in Becker muscular dystrophy and its potential as a disease-modifying therapy.
Experts discussed new biomarker data from a phase 1/2 study, presented at the 2026 MDA Conference, of the gene therapy SGT-003 in boys living with Duchenne muscular dystrophy.
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]
The child neurologist at Children’s Hospital of Philadelphia discussed the real-world safety data of givinostat use in patients with Duchenne muscular dystrophy at the 2026 MDA Conference. [WATCH TIME: 5 minutes]
Early BB-301 gene therapy data show durable swallowing gains and benign safety in OPMD dysphagia, hinting at disease modification.
A phase 1/2 study aims to assess the safety and tolerability of nivudirsen, also known as BMN 351, in single ascending and multiple ascending doses among patients living with Duchenne.
A neurologist at Georgetown University, MedStar Health, discussed phase 2/3 data presented at the 2026 MDA Conference that supports efgartigimod as a novel treatment for inflammatory myopathies. [WATCH TIME: 2 minutes]
A retrospective cohort study reported that myocardial fibrosis detected by cardiac MRI was associated with higher fatigue scores among women carriers of Duchenne or Becker muscular dystrophy.
At the 2026 MDA Conference, a neurologist at Nemours Children's Hospital discussed practical strategies for navigating insurance to ensure patients can access newly available neuromuscular therapies. [WATCH TIME: 3 minutes]
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.
A comparative analysis presented at the 2026 MDA Conference suggests the intrathecal gene therapy Itvisma may achieve similar motor outcomes to nusinersen and risdiplam in patients with spinal muscular atrophy.
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.
John Crowley, chief executive officer of the Biotechnology Innovation Organization, discusses the evolving biotechnology ecosystem, emerging therapeutic technologies, and ongoing policy challenges shaping the future of rare disease innovation.
SAFARI44 launches a phase 3 test of del-zota for DMD exon 44 skipping, tracking muscle function and dystrophin gains over 54 weeks.
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.
The president and chief executive officer of the Biotechnology Innovation Organization shared insights on his keynote speech delivered at MDA 2026, reflecting on the evolving rare disease ecosystem. [WATCH TIME: 3 minutes]
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.
The study design of the phase 3 STELLAR trials, presented at the 2026 MDA Conference, aims to investigate salanersen among presymptomatic infants with spinal muscular atrophy.
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.
Phase 2 CANYON data show sevasemten keeps LVEF steady and NT-proBNP stable in adults with Becker muscular dystrophy, hinting at heart protection.
