MDA Clinical & Scientific Conference

Barry Byrne, MD, PhD, Powell Gene Therapy Center at the University of Florida, discusses gene therapy considerations for pediatric patients and how newborn screening can influence outcomes for patients with Duchenne muscular dystrophy (DMD).

Patient-reported outcomes measures in generalized myasthenia gravis (gMG) are more important than ever, for both those treating and being treated for the chronic autoimmune neuromuscular disorder, to have a more nuanced understanding of experiences and difficulties.

These are data to week 26 on the monoclonal antibody and antineoplastic agent; data out to week 52 of the MINT trial will be presented in a late-breaking oral session at the upcoming American Academy of Neurology Annual Meeting.

One of the major highlights of the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference is that invaluable networking opportunities can help clinicians elevate their own best practices.

Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating neuromuscular diseases but also raised concerns about how potential funding cuts could impact this community.

Rozanolixizumab is a high-affinity humanized immunoglobulin G4 monoclonal antibody and Fc receptor blocker approved to treat anti–acetylcholine receptor– and anti–muscle-specific kinase–positive generalized myasthenia gravis (gMG) in adult patients; administration is subcutaneous and takes approximately 15 minutes.

Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows Duchenne muscular dystrophy (DMD) progression.

Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular atrophy (SMA) aged 2 to 17 years, who had previously been shut out of receiving gene therapy.

Tenacious efforts at every level, from the individual clinician to the hospital to the state to Congress, will be needed to make sure patients can access life-saving gene therapies for neuromuscular diseases.

Posters presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference highlight the complex care needs and physical activity barriers for individuals with myotonic dystrophy.

Medication costs are only a piece of the puzzle when clinicians think about health care access, explains Leigh Maria Ramos-Platt, MD.

Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that benefits in functional outcomes, gene expression, and muscle imaging persist 2 years after receiving the gene therapy.

Tania Gendron, PhD, speaks to the present challenges for translating biomarker discoveries to clinical practice and offers insights to how these can be overcome.

Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic advances in treating spinal muscular atrophy (SMA) are not uniformly making it into the hands of patients who could benefit.

Chronic inflammatory demyelinating polyneuropathy (CIDP) can have a substantial impact on patients everyday life and, consequently, put added stress on health care systems.

The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but also logistical hurdles and a need for complex discussions between clinicians and families.

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to the novel development of AAV gene therapy and its mechanism of action.

Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.

Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach adulthood thanks to transformative therapy advances.

Robert Califf, MD, former commissioner of the FDA, delivered a keynote address at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference that highlighted the enormous opportunities for progress in neuromuscular disease care amid a changing policy environment.

The 2025 Muscular Dystrophy Association Clinical & Scientific Conference, convening in Dallas, Texas, from March 16-18, will feature clinical updates, expert insights, and breaking trial findings that sum up to a new frontier of care for neuromuscular diseases.

Look ahead to this year's Muscular Dystrophy Association (MDA) meeting, which will feature discussions on the latest gene therapies, clinical trial data, policy considerations, and more in the realm of neuromuscular disease.