I’ve been reflecting on the disappointing outcomes and the reaction to Sarepta Therapeutics’ recently announced clinical ...
Duchenne muscular dystrophy (DMD) is an inherited X-linked degenerative muscular disorder predominantly caused by frame-disrupting mutations arising from gross rearrangements in the dystrophin gene. 1 ...
Rescue of dystrophic skeletal muscle in mdx and utrophin/dystrophin-deficient (dko) mouse models by reintroduction of dystrophin has validated gene therapy as a potential therapeutic approach for ...
CAMBRIDGE, MA--(Marketwired - Apr 15, 2013) - Sarepta Therapeutics, Inc. (NASDAQ: SRPT) today provided an update on its discussions with the U.S. Food and Drug Administration (FDA) regarding a ...
Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production. Studies of the new protein ...
Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window BOSTON -- A ...
WASHINGTON, Feb. 16, 2022 (GLOBE NEWSWIRE) -- The Global Dystrophin Market size is expected to reach USD 30.22 Billion by 2028, exhibiting a Compound Annual Growth Rate (CAGR) of 45.9% during the ...
Duchenne muscular dystrophy is caused by mutations in the dystrophin encoding DMD gene that disrupt the reading frame. Muscle fibers lacking dystrophin are more sensitive to damage and over time ...
People with Duchenne muscular dystrophy carry a mutation in the DMD gene coding for dystrophin, a protein crucial for proper muscle function. Scientists are working to treat the disease at its genetic ...
Duchenne muscular dystrophy has revealed a challenge to researchers as an immune reaction to the defective muscle protein, dystrophin, makes it difficult to strenghten patients' muscles. Duchenne ...
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Duchenne Gene Therapy: Why The FDA Has Tightened Rules Following Two Teen Deaths
The FDA has added a boxed warning to Sarepta’s Duchenne gene therapy Elevidys after two teens died from liver failure and has limited its use to ambulatory patients aged 4 and above. Learn what ...
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