Roche is launching a new global phase 3 trial for the controversial Duchenne muscular dystrophy gene therapy Elevidys in another push for European approval. Rather than relying on patient groups to ...
Mesoblast (MESO) added ~6% in the premarket on Wednesday after the FDA cleared its Investigational New Drug application that sought approval to conduct a study to test its cell therapy, Ryoncil, in ...
Roche (RHHBY) announced on Thursday that it is initiating a new Phase 3 trial for Elevidys, a treatment it developed with Sarepta Therapeutics (SRPT) for the muscle-wasting disorder. Duchenne muscular ...
Mast, who has reported extensively on rare diseases for four years, spoke with Debra and Hawken Miller more than six times over the last eight months and interviewed more than a dozen other advocates, ...
The stock of small-cap biotech Wave Life Sciences Inc. soared 51% Tuesday after the company reported positive interim data from a mid-stage trial of a treatment for the rare muscle disorder Duchenne ...
Theresa Gaffney is the lead Morning Rounds writer and reports on health care, new research, and public policy, with a particular interest in mental health, gender-affirming care, and LGBTQ+ patient ...
We have so many different fronts on which we face challenges in treating this disease. We have to start with equity and access. So many of our families face barriers to getting to care, whether it be ...
From inside Al Jalila Children’s Hospital in Dubai, Yousef Haider began his first steps in the journey of treatment for Duchenne muscular dystrophy, surrounded by the unbreakable heart of a mother and ...
Partnering with Parent Project Muscular Dystrophy to ensure timely access to the trial for eligible patients ~15,000 children are living with DMD in the U.S. NEW YORK, April 07, 2026 (GLOBE NEWSWIRE) ...
Sarepta Therapeutics (SRPT) shares fell after its late-stage trial for two gene therapies, AMONDYS 45 (casimersen) and VYONDYS 5 (golodirsen), aimed at treating Duchenne muscular dystrophy, did not ...
Duchenne muscular dystrophy (DMD) is a genetic disease that causes progressive muscle weakness. DMD commonly affects boys, where symptoms are seen in early childhood. There is currently no cure for ...
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