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 […]
– ENTR-601-44 was well-tolerated in healthy volunteers with no serious adverse events, no drug-related adverse events and no clinically significant changes or trends noted in vital signs, ECGs, physical exams or […]
REGENXBIO announced they are expanding their AFFINITY DUCHENNE Trial, which delivers a micro-dystrophin via AAV8, to include a new cohort of younger patients. The trial, which has been enrolling ambulatory […]
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 […]
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 […]
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 […]
Cranbury Pharmaceuticals Receives U.S. FDA Approval for First Generic Version of Emflaza® Oral Suspension (deflazacort) for Duchenne.
Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy.
Read the full news release HERE Capricor Therapeutics, which received early funding from CureDuchenne, has announced positive 3-year results from the HOPE-2 open-label extension study. Individuals receiving CAP-1002 continue to […]
