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240,588 Results for "muscular dystrophy association".
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Press Releases
Satellos Announces Upcoming Presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference
February 18, 2025
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6 min read
Policy
Muscular Dystrophy Association Applauds Expanded US FDA Approval of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy Patients Ages 4 and Above
The Muscular Dystrophy Association celebrates the U.S. Food and Drug Administration’s expanded approval of ELEVIDYS.
June 20, 2024
·
5 min read
Duchenne muscular dystrophy
Duchenne Muscular Dystrophy Space on Cusp of Pivotal Era
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several companies including Wave, Dyne and Avidity are looking to answer the call with investigational therapies targeting greater efficacy and broader reach.
January 27, 2025
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8 min read
·
Kate Goodwin
Press Releases
Scholar Rock to Present Additional Clinical Data from the Phase 3 SAPPHIRE Trial at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
February 21, 2025
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9 min read
Pharm Country
Muscular Dystrophy Association’s Funding of Foundational Research Leads to New FDA Approved Treatment Duvyzat (givinostat) for Duchenne Muscular Dystrophy
The Muscular Dystrophy Association’s funding of foundational research leads to new drug approval by the US Food and Drug Administration (FDA) of Duvyzat (givinostat), a histone deacetylase (HDAC) inhibitor to treat children and adolescents living with Duchenne muscular dystrophy (DMD).
March 21, 2024
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5 min read
Press Releases
Benitec Biopharma Announces Acceptance of Late- Breaking Oral Abstract for the BB-301 Phase 1b/2a Clinical Study at the Muscular Dystrophy Association Clinical and Scientific Conference
February 17, 2025
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6 min read
Press Releases
Santhera Announces Agreement with German GKV-SV on Reimbursement Amount for AGAMREE® (vamorolone) for the Treatment of Duchenne Muscular Dystrophy in Germany
February 13, 2025
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5 min read
FDA
Muscular Dystrophy Association-Supported Drug AGAMREE® (vamorolone) Approved for the Treatment of Duchenne Muscular Dystrophy by the FDA
The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of AGAMREE® (vamorolone), a structurally unique steroidal anti-inflammatory drug to treat children and adolescents living with Duchenne muscular dystrophy (DMD).
October 26, 2023
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6 min read
Press Releases
Santhera Receives Positive NICE Final Guidance for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy
January 16, 2025
·
4 min read
Drug Development
Constant Therapeutics Announces First Patient Dosed in Phase 2 Clinical Trial of TXA127 in Duchenne Muscular Dystrophy-Associated Cardiomyopathy
Constant Therapeutics LLC today announced that the first patient was dosed March 3, 2024, in the Company’s phase 2 clinical trial of TXA127, the Company’s lead peptide product under development as a potential treatment for Duchenne Muscular Dystrophy-associated (DMD-associated) Cardiomyopathy.
March 4, 2024
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2 min read
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