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Antisense Therapeutics Astellas Pharma Avidity Biosciences Catalyst Pharmaceuticals Dyne Therapeutics Edgewise Therapeutics Entrada Therapeutics FDA FibroGen Italfarmaco NS Pharma PepGen Pliant Therapeutics PTC Therapeutics REGENXBIO Santhera Sarepta Therapeutics Solid Biosciences Wave Life Sciences
April 17, 2024

Edgewise Therapeutics Announces Positive Two-Year Topline Results from the ARCH Open Label Trial of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy

Edgewise shares positive two-year topline results from the ARCH trial.
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April 1, 2024

Pfizer Launches Caregiver Educational Platform Reimagine Duchenne

Pfizer launches Duchenne caregiver education website.
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March 22, 2024

FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy

Jett Foundation is thrilled to share that the U.S. Food and Drug Administration (FDA) has approved Duvyzat (givinostat) to treat children and adolescents with Duchenne muscular dystrophy (DMD).  Please read...
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March 19, 2024

PTC Therapeutics Provides Key Regulatory Updates

PTC plans to re-submit an NDA for Translarna™ for the treatment of nonsense mutation Duchenne muscular dystrophy.
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March 13, 2024

Catalyst Pharmaceuticals Announces AGAMREE® Now Commercially Available in the U.S. for the Treatment of Duchenne Muscular Dystrophy

Following the FDA approval on October 26, 2023, AGAMREE is now available by prescription and dispensed throughout the United States through a specialty pharmacy network.
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March 13, 2024

PepGen Receives U.S. FDA Orphan Drug and Rare Pediatric Disease Designations for PGN-EDO51 for the Treatment of Duchenne Muscular Dystrophy

PepGen shares a press release announcing the FDA has granted both orphan drug and rare pediatric disease designations for PGN-EDO51.
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March 5, 2024

REGENXBIO Announces New Positive Initial Efficacy Data From Affinity Duchenne® Trial

REGENXBIO reports additional interim safety and efficacy data in the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 in patients with Duchenne.
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February 16, 2024

Sarepta Therapeutics Announces U.S. FDA Acceptance of an Efficacy Supplement to Expand the ELEVIDYS Indication

Sarepta announces the U.S. Food and Drug Administration (FDA) has accepted and filed the Company's efficacy supplement to the Biologics License Application for ELEVIDYS.
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February 13, 2024

Edgewise Receives U.S. FDA Fast Track Designation for EDG-5506 for the Treatment of Duchenne Muscular Dystrophy

Edgewise shares a press release announcing FDA granting Fast Track designation for EDG-5506 for the treatment of Duchenne.
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February 8, 2024

REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial

REGENXBIO announces completion of enrollment in the Phase I/II AFFINITY DUCHENNE® trial of RGX-202.
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