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Duchenne muscular dystrophy
Policy
FDA Accepts Sarepta’s Efficacy Supplement Seeking to Expand Elevidys Indication
The regulator has granted a priority review of the efficacy supplement for Sarepta Therapeutics’ gene therapy Elevidys with a target decision date of June 21, 2024.
February 16, 2024
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1 min read
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Tyler Patchen
PRESS RELEASES
Press Releases
Medera Receives FDA Fast Track Designation for Gene Therapy Targeting Duchenne Muscular Dystrophy-Associated Cardiomyopathy
April 16, 2026
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5 min read
Press Releases
Mesoblast Receives IND Clearance From FDA to Directly Proceed to Registrational Trial for Approval of Ryoncil® in Duchenne Muscular Dystrophy - CORRECTION
April 13, 2026
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8 min read
Press Releases
Atossa Therapeutics Reports Fourth Quarter and Year-End 2025 Financial Results and Provides a Corporate Update
March 26, 2026
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14 min read
Press Releases
PPMD Provides $250,000 in Funding to Secretome Therapeutics Through PPMD Venture Pathways Program to Support Development of Cardiac Cell Therapy
March 24, 2026
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4 min read
Press Releases
Sarepta Provides Regulatory Update on AMONDYS 45® and VYONDYS 53®
March 19, 2026
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15 min read
Press Releases
Atossa Therapeutics Strengthens Clinical Leadership Team with the Addition of Two Experienced Biopharma Executives
March 19, 2026
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6 min read
Press Releases
Sarepta Announces that Screening and Enrollment are Underway in ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duchenne
March 16, 2026
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11 min read
Press Releases
Capricor Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results and Provides Corporate Update
March 13, 2026
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14 min read
Press Releases
Capricor Therapeutics Announces Late-Breaking HOPE-3 Data at the 2026 MDA Conference Demonstrating Significant Functional Benefits of Deramiocel for Duchenne Muscular Dystrophy
March 13, 2026
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8 min read
Press Releases
Atossa Therapeutics Presents Clinical Trial Update Highlighting (Z)-Endoxifen Research at the 2026 MDA Clinical & Scientific Conference
March 12, 2026
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5 min read
Press Releases
Full Year 2025 Trading Update
March 12, 2026
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8 min read
Press Releases
Gene Therapy for Duchenne Muscular Dystrophy: Genethon Confirms Two-Year Efficacy in Patients Treated with Its Drug Candidate GNT0004 at Therapeutic Dose in the First Phase of Its Clinical Trial
March 11, 2026
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4 min read
Press Releases
Capricor Therapeutics Announces Establishment of New PDUFA Date for Deramiocel BLA
March 10, 2026
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6 min read
Press Releases
Precision BioSciences Presents Preclinical PBGENE-DMD Data Highlighting Durable Dystrophin Expression and Functional Benefit at the Muscular Dystrophy Association Clinical & Scientific Conference 2026
March 10, 2026
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7 min read
Press Releases
Satellos Presents Interim SAT-3247 Clinical and Biomarker Data in Duchenne Muscular Dystrophy at the 2026 MDA Clinical & Scientific Conference
March 10, 2026
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7 min read
Press Releases
Parent Project Muscular Dystrophy, Foundation to Eradicate Duchenne Announce 2026 Advocacy Leadership Awards Recipients Honoring Bipartisan Champions in Congress
March 9, 2026
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3 min read
Press Releases
Precision BioSciences Receives FDA Fast Track Designation for PBGENE-DMD and Announces Duchenne Muscular Dystrophy Investor Event
March 9, 2026
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6 min read
Press Releases
Tenaya Therapeutics Presents Preclinical Data at MDA 2026 Highlighting TN-301’s Potential to Correct Skeletal and Cardiac Muscle Decline in Duchenne Muscular Dystrophy
March 9, 2026
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8 min read
Press Releases
ITF Therapeutics LLC Presents New Data and Analyses on Givinostat for Treatment of Duchenne Muscular Dystrophy at 2026 MDA Clinical and Scientific Conference
March 9, 2026
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9 min read
Press Releases
Keros Therapeutics Presents Additional Clinical Data from Its Rinvatercept Program at the 2026 MDA Clinical & Scientific Conference
March 9, 2026
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6 min read
