A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular dystrophy, according to new research from the Smidt Heart Institute at ...
By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...
Werner is CEO of Alltrna and CEO-partner at Flagship Pioneering. Duchenne muscular dystrophy has seen more progress in the past 15 months (give or take) than in the last couple of decades combined. In ...
Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
A patient has died after receiving Sarepta Therapeutics’ approved gene therapy for Duchenne muscular dystrophy — the second fatality reported by the company in the past three months. Like the first ...
To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of ...
On a Monday conference call, Sarepta CEO Doug Ingram said new long-term data should help “rebalance the discussion” surrounding Elevidys.
The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication. The ...
A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said in a statement ...
When you're diagnosing a child with Duchenne muscular dystrophy, says Alexandra Bonner, MD, of Cleveland Clinic, it's important to not only determine if she's a carrier of the gene mutation that ...
Duchenne muscular dystrophy (DMD) is a genetic condition that causes increasing muscle weakness over time. While DMD currently has no cure, researchers are studying potential new treatments. When ...
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