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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 […]
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.
Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy
Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy.
Capricor Therapeutics Announces Positive 3-Year Efficacy Results from HOPE-2 Open Label Extension Study of CAP-1002 in 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 […]
hC Bioscience Announces Program in Duchenne Muscular Dystrophy and Reports In Vivo Data From tRNA-based Protein Editing Platform
As a funder of hC Bioscience, we’re happy to have them announcing their tRNA-based approach for targeting nonsense mutations at our annual Futures conference.
Dyne Therapeutics Announces New Clinical Data from ACHIEVE Trial of DYNE-101 in DM1 and DELIVER Trial of DYNE-251 in DMD Demonstrating Compelling Impact on Key Disease Biomarkers and Improvement in Multiple Functional Endpoints
As early investors in Dyne Therapeutics, we are pleased to share they reported positive updates in their next-generation exon skipping program for Duchenne amenable to skipping exon 51. In their […]