Muscular Dystrophy Association Conference

All three patients experienced improvements in various aspects of muscle function, heart and lung health, and daily movement.

Delandistrogene moxeparvovec is designed to counter muscle breakdown by delivering dystrophin to muscle cells.

Better mobility and sustained functional dystrophin levels were seen in young boys with DMD treated with delandistrogene moxeparvovec.

Without a structured transition plan, young adults with neuromuscular diseases may face significant risks.

Emerging genetic testing technologies include optical genome mapping, long-read sequencing, and RNA sequencing.

Scientists compared growth velocities in patients with DMD treated with vamorolone, prednisone, deflazacort, or placebo.

Patients who started corticosteroids at a younger age had a lower incidence of depressive symptoms.

“Even with the same disease state, the phenotype is very different,” and should influence treatment strategies, an expert said.

Only 4 states have approved newborn screening for Duchenne muscular dystrophy.

The federal Department of Transportation in 2024 finalized a rule that enhances air travel experiences for wheelchair users.