Jeff Erlanger was a longtime fan of Fred Rogers before appearing on his show, and the two became friends for life ...

SAN DIEGO — Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory boys with Duchenne muscular dystrophy, interim data show.

A recent discovery of a molecular connection between autism and myotonic dystrophy, a type of neuromuscular disease, may provide a breakthrough on how clinicians approach autism spectrum disorder.

“There are no disease-modifying treatments approved for patients with any subtype of limb-girdle muscular dystrophy, and the unmet medical need is significant. Following feedback from U.S ...

Sarepta (SRPT) shared updates from its clinical programs focused on limb-girdle muscular dystrophy subtypes 2C/R5, 2D/R3, and 2E/R4. Following input from the FDA, Office of Therapeutic Products ...

Sarepta Therapeutics SRPT announced updates from its clinical programs, which focus on targeting different subtypes of limb-girdle muscular dystrophy (LGMD), a group of rare genetic disorders ...

Pfizer has started phase II trials of a candidate drug to treat Duchenne muscular dystrophy (DMD) as it plays catch-up with rivals PTC Therapeutics, Prosensa and Sarepta. The pharma giant has ...

How a 1981 episode of 'Mister Rogers' Neighborhood' opened kids' eyes to one boy's story — and now inspires a new character ...

Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy, the biotech has closed a public offering that raised an impressive ...

Deflazacort is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older. The group now has 424 approvals and has so far filed 492 ...

This drug helps treat Duchenne muscular dystrophy (DMD) — a rare condition that causes muscle weakness and degeneration, often seen in children. The company can now launch a generic version of ...