The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment's ability to help ...

Highlights from the 2025 Muscular Dystrophy Association (MDA) Conference include clinical updates, expert insights, and ...

Catalyst is offering patients and their caregivers a personalized program called Catalyst Pathways to provide guidance and financial support. Agamree ® (vamorolone) oral suspension has been made ...

This is an archived article and the information in the article may be outdated. Please look at the time stamp on the story to see when it was last updated. There’s news of a potential treatment that ...

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window Eight therapies ...

Experts discuss the landscape of treatment options for Duchenne muscular dystrophy, examine the benefits and risks of gene therapy and conventional steroid treatments, and emphasize the need for early ...

In an eagerly anticipated decision, the Food and Drug Administration Thursday approved the first gene therapy for muscular dystrophy. "Today's approval addresses an urgent unmet medical need and is an ...

BELIEVE WHAT HAPPENED NEXT. FOUR YEAR OLD LUCAS LOVES A GOOD PLAYDATE AT THE PARK. HI, LUCAS. HI. IT’S SANDBOX TRUCKS TODAY, BUT HIS FAVORITE PLAYING SUPERHEROES. THIS IS HIS FAVORITE RIGHT NOW. PJ ...

HOUSTON – There’s a new type of therapy that can help kids with Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) affects one in 3,600 male births, and it can lead to immobility, and ...

Dyne Therapeutics’ experimental treatment for Duchenne muscular dystrophy (DMD) showed promise in new data from a small Phase 1/2 trial dubbed DELIVER, the pharma announced this week. The drug, ...

The problems began sometime before JB turned 2. He was, by appearance, a healthy boy. He had a brilliant smile, striking blonde hair and eyes the color of the sea. He fast was becoming enamored with ...