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.

“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 ...

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule.

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 ...

Alison Becker acknowledges that it is becoming harder to avoid talking about how close Liverpool are to winning the Premier League title. Slot and his team now need only two more wins to ...

The effects of the Trump administration’s decision to freeze more than $2.2 billion in federal funding to Cambridge, Mass.-based Harvard University are coming into sharper focus, as researchers ...

West Ham had beaten Liverpool's offside trap. But not their goalkeeper. Alisson Becker saved. Bowen became his latest victim. Just another example of why the Brazilian is the best in those moments.

“Budgetary issues are always a concern, whether it’s at the national level and the state level,” Dr. Antonucci told Becker’s in April. “We are in the middle of our legislative session ...

Welcome to the Bermuda Dollar exchange rate & live currency converter page. The Bermuda Dollar (BMD) exchange rates represented on this page are live, updated every minute within the forex market ...

Despite gastroenterologist pay falling by 3% between 2024 and 2025, overall, the specialty has seen a 33.78% pay hike since 2015. From 2015 to 2025, the inflation rate in the U.S. has risen by 1.7%, ...