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237,751 Results for "muscular dystrophy association".
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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
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5 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
Biotech Beach
Capricor Therapeutics Announces for the Treatment of Duchenne Muscular Dystrophy
Capricor Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA (Biologics License Application) meeting with the Company in the third quarter of 2024 for deramiocel (CAP-1002), for the treatment of Duchenne Muscular Dystrophy (DMD).
June 25, 2024
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5 min read
Press Releases
Genethon Presents Positive Initial Results from a Phase 1/2/3 Trial of its Gene Therapy (GNT0004) for Duchenne Muscular Dystrophy at ASGCT Breakthroughs in Muscular Dystrophy in Chicago
November 19, 2024
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5 min read
Positive Results from Genethon’s Gene Therapy Trial for Duchenne Muscular Dystrophy, Advances of Its Spinoff Atamyo in the LGMD and Cardiomyopathy Fields, Highlighted in Latest Newsletter
Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association, provided updates in its latest Newsletter on results of its gene therapy clinical trials for Duchenne Muscular Dystrophy and advances in gene therapies for limb-girdle muscular dystrophies by its spinoff Atamyo Therapeutics.
June 3, 2024
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2 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
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
Drug Development
Pfizer’s Gene Therapy for Duchenne Muscular Dystrophy Fails Phase III Trial
Pfizer’s investigational Duchenne muscular dystrophy gene therapy, fordadistrogene movaparvovec, failed in a late-stage study to significantly improve motor function in patients versus placebo.
June 13, 2024
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2 min read
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Tristan Manalac
BioCapital
Elixirgen Therapeutics Presents Preclinical Data with Bobcat mRNATM Technology in Duchenne Muscular Dystrophy at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
Elixirgen Therapeutics, Inc., today presented preclinical data on its proprietary Bobcat mRNA TM technology in a poster presentation at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference taking place March 3-6, 2024, in Orlando, Florida.
March 4, 2024
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2 min read
FDA
FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy
Today, the U.S. Food and Drug Administration expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) for ambulatory and non-ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene.
June 20, 2024
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8 min read
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