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 ...
Highlights from the 2025 Muscular Dystrophy Association (MDA) Conference include clinical updates, expert insights, 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 ...
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 ...
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 ...
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 ...
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 ...
SAN DIEGO, July 23, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide ...
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