By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...
Sarepta’s blockbuster gene therapy has come under scrutiny following a string of patient deaths.
After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing ...
In his CEO Commentary, Frederic Revah, Ph.D., underscores the dedication, expertise and successes of Genethon's 240 scientists and professional staff as they pursue new gene therapy treatments for ...
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on ...
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced ...
In research published in Developmental Medicine & Child Neurology, investigators have developed a brief, reliable, and valid ...
Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE, /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa ...
EPIDYS trial met primary endpoint demonstrating statistically and clinically meaningful treatment benefit in one of the largest DMD phase 3 trials to date Median follow-up of over 3 additional years ...
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 ...
"Our study is one of very few that shows a relationship between prophylactic cardiac medication use and survival in DMD…This is a strong addition to the existing literature supporting early initiation ...
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