Tag: Duchenne Muscular Dystrophy

Precision BioSciences Earns FDA Orphan Drug Status for Duchenne Muscular Dystrophy

July 23, 2025

Precision BioSciences has been given Orphan Drug Designation by the U.S. Food and Drug Administration for its new treatment, PBGENE DMD, which is being developed

Precision Bio Gets FDA Rare Disease Nod for DMD Therapy

June 25, 2025

Precision BioSciences, a clinical stage gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for diseases with high unmet need,

Sarepta Updates UK Dosing in ENVISION Study of ELEVIDYS

May 21, 2025

Sarepta Therapeutics, the leader in precision genetic medicine for rare diseases, shared the following update related to ELEVIDYS, the only approved gene therapy for patients

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Tag: Duchenne Muscular Dystrophy

Precision BioSciences Earns FDA Orphan Drug Status for Duchenne Muscular Dystrophy

Precision Bio Gets FDA Rare Disease Nod for DMD Therapy

Sarepta Updates UK Dosing in ENVISION Study of ELEVIDYS

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