ICYMI: Highlights From MDA 2025

The 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which convened in Dallas, Texas, from March 16-18, featured clinical updates, expert insights, and breaking trial findings that sum up to a new frontier of care for neuromuscular diseases. Researchers, clinicians, policy leaders, and patient advocates came together to discuss the state of neuromuscular disease care, gene therapy, and access while celebrating 75 years of the MDA.

Below are the 5 most-read AJMC.com pieces from the conference; you can also catch up on all our coverage on our dedicated MDA page.

A proliferation of gene therapies and novel neuromuscular treatments brings hope but also challenges for clinicians, health systems, and families | Image Credit: © anna - stock.adobe.com

5. Building Community and Advancing Care at MDA 2025

This recap video captures the importance of the conference as a chance for networking, mentorship, collaboration, and cross-pollination of ideas among researchers, clinicians, and advocates. Attendees, including Sharon Hesterlee, PhD, then the chief research officer at MDA and now the president and CEO, and Leigh Maria Ramos-Platt, MD, clinical professor of neurology and pediatrics at USC Keck School of Medicine, emphasized that the informal interactions often catalyze new research collaborations or operational improvements in neuromuscular disease care. Especially in a rapidly evolving therapeutic landscape, having a community that shares best practices, supports each other, and builds consensus around care standards is critical.

Watch the full video.

4. Neurologists Share Tips for Securing Patient Access to Gene Therapies

A recurring theme at MDA 2025 was that exciting scientific advances only matter if patients can actually receive them, so access remains a major hurdle. This article covers a session that delivered practical tips from clinicians and advocates on navigating payer coverage, prior authorization, regulatory barriers, and coordinating multidisciplinary care to ensure patients receive gene therapies when indicated. The speakers’ insights underscored that across levels—from individual doctors to hospitals, insurers, and policy makers—persistence is needed to convert gene therapy breakthroughs into real-world benefit. “The science is on our side…in terms of treating these kids,” said Lamar Davis II, MD, associate professor of pediatrics at the University of Mississippi Medical Center. “We just have to be willing to put in work and not stop.”

Read the full article.

3. STEER Data Open Door to SMA Gene Therapy for Wider Age Range of Children

One of the most important scientific takeaways from MDA 2025 was the phase 3 STEER study of onasemnogene abeparvovec (Zolgensma; Novartis), which showed that intrathecal delivery in children aged 2 to 17 years with spinal muscular atrophy (SMA) was safe and yielded statistically significant motor function improvements compared with a sham procedure. This expands the potential reach of gene therapy beyond infants to children older than 2, who previously had limited treatment options, and could meaningfully improve long-term outcomes for the broader SMA population. Just 8 months after the data were presented at MDA 2025, the FDA approved onasemnogene abeparvovec-brve (Itvisma; Novartis) for intrathecal delivery in adults and children 2 years and older with confirmed SMN1 mutations, marking the first time a gene replacement therapy is available to this broad population.

Read the full article.

2. How Access to SMA Treatment Varies Globally and by Insurance Type

Despite these therapeutic advances, multiple posters at MDA 2025 revealed that access to SMA treatments remains uneven. Two covered in this write-up showed that patients’ likelihood of receiving new SMA therapies depends heavily on geographic location, age, and type of insurance coverage. Although newborn screening for SMA is nearly universal in the US, globally only a small fraction of newborns benefit from such programs—limiting early detection and timely treatment. Moreover, even within insured populations, denials are common: employer-based plans appeared more likely to deny coverage, with resulting appeals processes delaying therapy by weeks, which could have real clinical consequences. The findings spotlight a persistent equity and access gap, as breakthroughs in therapy mean little if distribution and insurance coverage do not keep pace.

Read the full article.

1. Bustling Gene Therapy Pipeline for Neuromuscular Diseases Brings Thorny Questions to the Clinic

Topping the 2025 MDA roundup, this article summarized a session full of tension and excitement, as clinicians are witnessing a proliferation of gene therapies and novel neuromuscular treatments that bring hope but also challenges for clinicians, health systems, and families. This session covered the practical considerations around gene therapies for neuromuscular diseases, including SMA and Duchenne muscular dystrophy. Speakers and panelists emphasized that as therapeutic options expand, clinics must build capacity—not just in infusion or intrathecal delivery, but also in long-term monitoring, patient support, and care coordination. Payers, regulators, and advocacy groups must work together to ensure equitable access and sustainable funding and that real-world outcomes are tracked thoroughly, they agreed.

Read the full article.