Neuromuscular disease researchers, clinicians, and advocates gathered to celebrate 75 years of progress at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Dallas, Texas, but they also noted that these gains could be reversed if cuts to Medicaid and […]
MDA Reflects on 75 Years of Research and the Precarious Times Ahead Read More »
Results from the phase 3 STEER study of intrathecal onasemnogene abeparvovec showed encouraging efficacy and safety data for children aged 2 to 17 years with spinal muscular atrophy (SMA), paving the way for much broader uptake of this disease-modifying gene
STEER Data Open Door to SMA Gene Therapy for Wider Age Range of Children Read More »
Transformative gene therapies for neuromuscular diseases won’t make it into patients’ hands without the tenacious efforts of clinicians, hospital administrators, and other advocates, experts said at a session of the 2025 Muscular Dystrophy Association Clinical & Scientific Conference in Dallas,
Neurologists Share Tips for Securing Patient Access to Gene Therapies Read More »
Encouraging long-term outcomes were seen in patients with Duchenne muscular dystrophy (DMD) who received the gene therapy delandistrogene moxeparvovec (Elevidys; Sarepta) in the EMBARK trial, according to findings presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
EMBARK Data Show Continued Improvements With DMD Gene Therapy Read More »
Newly approved therapies for spinal muscular atrophy (SMA) hold the potential to modify the disease course and extend patients’ life spans, but research shows that patients have variable likelihoods of receiving these therapies based on their country of residence, age,
How Access to SMA Treatment Varies Globally and by Insurance Type Read More »
As the number of gene therapy options to treat neuromuscular diseases like Duchenne muscular dystrophy (DMD) and spinal muscular atrophy grows, so will the need to strengthen the delivery infrastructure, understand the risk-benefit calculus, and gather data across age groups,
As gene therapies transform the care of muscular dystrophy, more and more patients are surviving with these diseases that used to kill patients before they reached adulthood. This influx of adults into the care delivery system is a great “problem”
Between gene editing capabilities, artificial intelligence, platform technology to speed drug delivery, and the talent and dedication of those working in the neuromuscular disease field, “these are tremendous opportunities—there’s never been a better time—but we have enormous headwinds at the
For Neuromuscular Disease Community, an Era of Opportunities and Threats Read More »
In a hearing of the Senate Finance Committee on his nomination to serve as administrator of CMS, Mehmet Oz, MD, faced questions on the looming potential of cuts to Medicaid but mainly emphasized the role of nutrition and lifestyle in
Oz Confirmation Hearing Probes Vision for Medicaid but Coalesces Around Well-Being Read More »
When the Muscular Dystrophy Association (MDA) was founded in 1950, research on this group of disorders was limited—and treatment options even more so.1 In the years that followed, advocacy efforts by stars like Jerry Lewis drew attention and funding via
Regenerating Neurons, Muscle, and Hope in the Field of Muscular Dystrophy Read More »
