Posters presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference highlight the complex care ...

Myotonic dystrophy type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely ...

Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, ...

ROCKVILLE, MD, USA I March 19, 2025 I REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase ...

Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach ...

RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...

We all have different situations, stories, reasons why we need to travel differently,” said one traveler. And if we don’t now ...

Taking her family’s struggle with a terminal illness and creating a non-profit to help others going through the same ...

Sarepta Therapeutics (SRPT) stock drops 25% as the company reports a patient death linked to Elevidys, its gene therapy developed with Roche (RHHBY). Read more here.

Q4 2024 Earnings Call Transcript March 13, 2025 REGENXBIO Inc. beats earnings expectations. Reported EPS is $-1.01, ...

Beyond financials, the business is advancing its drug pipeline, with early clinical data for BMN 351 (Duchenne Muscular Dystrophy) and BMN 333 expected later this year. These developments could ...

RGX-202 is the only microdystrophin construct to include the C-terminal domain ... as a best-in-class gene therapy for Duchenne muscular dystrophy. We look forward to sharing additional biomarker ...