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Edgewise Therapeutics announced the launch of an educational website dedicated to Becker muscular dystrophy
Edgewise Therapeutics announced the launch of an educational website dedicated to Becker muscular dystrophy
Avidity Biosciences Reports Positive Data Demonstrating AOC 1044 Delivers Unprecedented Concentrations of PMO in Muscle Following a Single Dose in Healthy Volunteers from Phase 1/2 EXPLORE44™ Trial for Duchenne Muscular Dystrophy
As an early funder of Avidity Biosciences, we are pleased to share that AOC 1044, designed to skip exon 44 in the dystrophin gene, produced significant exon skipping in healthy volunteers.
Capricor Therapeutics Announces Continuation of Phase 3 HOPE-3 Trial
Capricor Therapeutics Announces Continuation of Phase 3 HOPE-3 Trial of CAP-1002 in Duchenne Muscular Dystrophy Based on Completion of Interim Futility Analysis
Three Things You Missed From the AGAMREE (Vamorolone) Webinar
Agamree (vamorolone) was recently approved by the FDA, and Catalyst explains the new steroid and how to get it in this CureDuchenne Webinar.
Astellas study results are available for ASP0367 (MA-0211) in Pediatric Male patients with Duchenne Muscular Dystrophy (0367-CL-0102)
Read Astellas community letter below:
Solid Biosciences received clearance by the U.S. FDA for an Investigational New Drug (IND) application for SGT-003 for DMD
Solid Biosciences Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003
CureDuchenne Raises More Than $1.3 Million at Ninth Annual Napa in Newport to Help Find a Cure for Duchenne Muscular Dystrophy
Southern California’s Premier Wine Event Raised Funds and Awareness for Rare Disease with Acclaimed Napa Valley Vintners. NEWPORT BEACH Calif., November 14, 2023 – CureDuchenne and Vintner Chair Melka Estates hosted the Ninth Annual Napa […]
CureDuchenne Welcomes New Executive Director, Vice President of Medical Affairs, and Board Members
Leading Duchenne Muscular Dystrophy Nonprofit Strengthens Leadership Team and Board Expertise
Santhera Receives Positive CHMP Opinion Recommending Approval of AGAMREE® (vamorolone) for the Treatment of Duchenne Muscular Dystrophy
The Committee for Medicinal Products for Human Use (CHMP) has given a positive recommendation for AGAMREE’s approval in Europe.