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 ...

This is an archived article and the information in the article may be outdated.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 ...

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 ...

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 ...

Scientists have developed a novel approach of genome editing to repair muscle stem (satellite) cells, offering new hope for ...

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 ...

When Conner Curran was first diagnosed with Duchenne muscular dystrophy at age 4, his parents were told there was nothing they could do to change his life's course. Jessica and Chris should enjoy ...

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 ...

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, ...

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 ...

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 ...