Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...
Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new ...
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Sarepta pops on Elevidys data; but will it save the embattled gene therapy?
Sarepta stock jumped Monday after the company unveiled three-year test results for its embattled gene therapy, Elevidys.
In human biology, the protein dystrophin is a shining example of Joni Mitchell’s classic line, “you don’t know what you’ve got ’til it’s gone.” Dystrophin stabilizes muscle cells. In its absence, the ...
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 ...
Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...
Avidity Biosciences, Inc. (NASDAQ:RNA) on Wednesday shared new data from participants treated continuously with del-zota for one year in the EXPLORE44 and EXPLORE44-OLE trials for Duchenne muscular ...
Dyne Therapeutics’ experimental treatment for Duchenne muscular dystrophy (DMD) showed promise in new data from a small Phase 1/2 trial dubbed DELIVER, the pharma announced this week. The drug, ...
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 ...
SEATTLE, Nov. 17, 2025 /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa" or the "Company"), a clinical-stage biopharmaceutical company developing innovative medicines in oncology, ...
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