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REGENXBIO Announces Expansion of AFFINITY DUCHENNE® Trial to Include New Cohort of Younger Patients
REGENXBIO has announced it initiated enrollment in a new cohort of patients ages 1-3 in its Phase I/II AFFINITY DUCHENNE® trial. The trial is evaluating the safety and efficacy of RGX-202, an investigational gene therapy that utilizes a novel adeno-associated virus (AAV8) to transport a shortened version of the dystrophin gene (micro-dystrophin) that may provide…
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Genethon’s Duchenne Muscular Dystrophy Gene Therapy GNT0004 Produces Expression of Microdystrophin in Phase 1/2/3 Trial
Genethon reported positive results from its AAV-microdystrophin treatment trial in Europe. The three participants receiving the study’s higher dose (3×1013vg/kg) showed that an average of 54% of muscle fibers were producing microdystrophin, and individuals demonstrated a reduced level of creatine kinase, a marker of muscle damage. Genethon said it is planning a pivotal trial in…
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FSHD treatment del-brax (formerly AOC 1020) aids muscle function
AOC 1020, an investigational treatment by Avidity Biosciences that’s now called delpacibart braxlosiran, or del-brax, helped adults with facioscapulohumeral muscular dystrophy (FSHD) grow stronger muscles and extend their arms farther than they could four months earlier. These are early data from FORTITUDE (NCT05747924), a three-part Phase 1/2 clinical trial that’s testing how safe del-brax is…
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Elevidys DMD gene therapy now FDA-approved for ages 4 and older
The U.S. Food and Drug Administration (FDA) has expanded its approval of Elevidys (delandistrogene moxeparvovec-rokl), authorizing the one-time gene therapy for individuals with Duchenne muscular dystrophy (DMD) ages 4 and older regardless of their ability to walk. Previously, the treatment had been approved for DMD children ages 4 to 5 who could walk. Doug Ingram,…
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Finding a balance between fighting for healthcare and celebrating wins
Recently, our family had terrific reasons to celebrate. I have three sons with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15, and Charlie, 13. They are doing things we never imagined they’d do when they were young. Max, who recently graduated from high school, was in “The SpongeBob Musical” this spring. The cast was selected…
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La Organización Mundial Sin Fines de Lucro CureDuchenne Llevará a Cabo Seminarios Web Informativos Tras la Ampliación de la Etiqueta de ELEVIDYS, la Terapia Génica de Sarepta Therapeutics, por la FDA
NEWPORT BEACH, California, 21 de junio de 2024 /PRNewswire-HISPANIC PR WIRE/ — CureDuchenne, una organización mundial sin fines de lucro comprometida con la búsqueda y el financiamiento de una cura para la distrofia muscular de Duchenne, llevará a cabo seminarios web en inglés y español para la comunidad de Duchenne después de que la Administración de Alimentos y…
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Global Nonprofit CureDuchenne to Host Informational Webinars Following FDA Label Expansion of Sarepta Therapeutics’ Gene Therapy, ELEVIDYS
NEWPORT BEACH, Calif., June 20, 2024 /PRNewswire/ — CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy, will host webinars in English and Spanish for the Duchenne community after the U.S. Food and Drug Administration (FDA) approved the expansion to the label indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) from Sarepta Therapeutics to include individuals with…
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PAAC Travel Tips
Anyone living with Duchenne muscular dystrophy knows that there are extra steps to EVERYTHING. Traveling with medical equipment requires planning and organization. PPMD’s Adult Advisory Committee (PAAC) has put together several resources with tips that may be helpful for you when planning a trip by ground or air. Whether you are flying with a power…
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Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above
See the previous update from June 22, 2023 here FDA Grants Traditional Approval for Ambulatory Individuals and Accelerated Approval for Non-Ambulatory Individuals to Sarepta Therapeutics’ ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy We are pleased to share that Sarepta Therapeutics’ gene therapy for Duchenne, ELEVIDYS, has been granted traditional approval for ambulatory individuals and accelerated…
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FDA Expands ELEVIDYS Label Indication for Ages 4 Years and Older
PPMD is excited to share that the FDA has made the decision to expand the labeled indication for ELEVIDYS, a micro-dystrophin gene therapy used to treat people with Duchenne muscular dystrophy, to include individuals at least 4 years of age and remove ambulation restrictions. ELEVIDYS is made available and marketed in the United States by…
