An 11-year-old girl with muscular dystrophy was given a cheer as she walked down the hallway of Southampton Hospital as part ...

Stanford Medicine researchers found that a smartphone could monitor patients with two types of muscular dystrophy as well as ...

A long-running confirmatory trial of two of Sarepta Therapeutics’ Duchenne muscular dystrophy drugs has missed its main goal.

Sarepta said on Monday that its late-stage study testing two gene-targeted therapies for Duchenne muscular dystrophy did not ...

An 11-year-old girl with muscular dystrophy was given a cheer as she walked down the hallway of Southampton Hospital as part of an inspiring challenge.

Because researchers have made such striking progress in developing drugs to treat neuromuscular diseases, Scott Delp, Ph.D., ...

This novel therapy offers the first FDA-approved option to address the root cause of this ultra-rare condition, which causes progressive muscle weakness impairing walking, breathing, and swallowing.

An Omaha woman battling a disabling disease says she had been caught in the middle of a billing dispute. Notices from a ...

That research led to the FDA granting compassionate use of the doxecitine and doxribtimine treatment in Arturito Estopinan, ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the third quarter of 2025 and the completion of ESSENCE, its ...

Publication highlights Deramiocel’s anti-fibrotic activity and describes a validated potency assay supporting quality control and product consistency in late-stage development- ...

I just want to get my independence back” - the words of University of Sunderland student Melanie Hartshorn who needs to raise over £60,000 to get the pioneering spinal surgery that can save her life.