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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 […]
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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 […]
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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, […]
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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), […]
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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 […]
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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 […]
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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 […]
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PAAC Travel Tips
Anyone living with Duchenne muscular dystrophy knows that there are extra steps to EVERYTHING. Traveling […]
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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 […]
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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 […]
