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ITF THERAPEUTICS| DUVYZAT Update
CureDuchenne Webinar | ITF THERAPEUTICS Patient Community Update: An overview of DUVYZAT Recorded on June 26, 2024Watch HERE
Seminario Web Cure Duchenne: Explicación de la Decisión de la FDA acerca de Elevidys
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FDA APPROVAL OF ELEVIDYS
This webinar discusses a groundbreaking gene therapy treatment for Duchenne Muscular Dystrophy (DMD), focusing on a method to introduce a miniaturized version of the dystrophin gene using a viral vector. […]
Protected: Duchenne Bone Health Study
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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 […]
Entrada Therapeutics Reports Positive Preliminary Data in Healthy Volunteers from Phase 1 ENTR-601-44-101 Trial for Duchenne Muscular Dystrophy
– 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 Announces Expansion of AFFINITY DUCHENNE® Trial to Include a New Cohort of Younger Patients
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 […]
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 […]
Cranbury Pharmaceuticals Receives U.S. FDA Approval for First Generic Version of Emflaza® Oral Suspension (deflazacort) for Duchenne Muscular Dystrophy
Cranbury Pharmaceuticals Receives U.S. FDA Approval for First Generic Version of Emflaza® Oral Suspension (deflazacort) for Duchenne.