The FDA expanded the approval of delandistrogene moxeparvovec-rokl (Elevidys) gene therapy for Duchenne muscular dystrophy on Thursday to include ambulatory or non-ambulatory patients ages 4 years and ...
Please provide your email address to receive an email when new articles are posted on . The FDA has announced approval of a label expansion for Elevidys to include patients with Duchenne muscular ...
Highlights from the 2025 Muscular Dystrophy Association (MDA) Conference include clinical updates, expert insights, and ...
A Sarepta Therapeutics gene therapy that failed its confirmatory test has now converted its accelerated FDA approval into a traditional one, expanding use of the treatment to a wider group of Duchenne ...
Please provide your email address to receive an email when new articles are posted on . AOC 1044 demonstrated increased exon 44 skipping compared with placebo in an ongoing phase 1/2 trial. Avidity ...
(RTTNews) - Sarepta Therapeutics Inc. (SRPT) announced U.S. Food and Drug Administration approval of an expansion to the labeled indication for Elevidys (delandistrogene moxeparvovec-rokl) to include ...
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement: Just before 6:00 p.m. ET ...
Certain genetic mutations in patients with DMD influence the timing of ambulation loss, with exon 44 skipping, exons 3 through 7 deletions, and exon 2 duplications linked to delayed loss. The study's ...
Italfarmaco & JCR Pharma enter commercialisation agreement for givinostat to treat Duchenne muscular dystrophy in Japan: Milan, Italy Monday, December 29, 2025, 14:00 Hrs [IST] It ...
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