Precision BioSciences Receives U.S. FDA Clearance of Investigational New Drug Application for First-in-Class PBGENE-DMD for Treatment of Duchenne Muscular Dy…

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...

Duchenne Progress Shows How the FDA Should Judge Orphan Drugs

A milestone in the treatment of Duchenne muscular dystrophy (DMD) passed recently with remarkably little public notice. That ...
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FDA expands approval of first gene therapy for rare form of muscular dystrophy

The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the risks and benefits of the drug The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the ...

Study links lower HDAC11 to reduced muscle damage in Duchenne dystrophy mice

A preclinical study led by the Germans Trias i Pujol Research Institute (IGTP), in collaboration with the Institut de Myologie and the Sant Pau Research Institute, has analyzed the role of the protein ...

Atossa Therapeutics Maintains Strong Market Position for (Z)-Endoxifen for Duchenne Muscular Dystrophy as Congress Reauthorizes Priority Review Voucher Program

Atossa's Rare Pediatric Disease Designation for (Z)-endoxifen in neuromuscular diseases qualifies for a future PRV upon FDA approval SEATTLE, Feb. 5, 2026 /PRNewswire/ -- Atossa Therapeutics, Inc.
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FDA expands approval of first gene therapy for rare form of muscular dystrophy

The U.S. Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...