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 of CAP-1002 in Duchenne Muscular Dystrophy Based on Completion of Interim Futility Analysis
Agamree (vamorolone) was recently approved by the FDA, and Catalyst explains the new steroid and how to get it in this CureDuchenne Webinar.
Read Astellas community letter below:
Solid Biosciences Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003
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 […]
Leading Duchenne Muscular Dystrophy Nonprofit Strengthens Leadership Team and Board Expertise
The Committee for Medicinal Products for Human Use (CHMP) has given a positive recommendation for AGAMREE’s approval in Europe.
– GRAND CANYON is the first pivotal study of an investigational therapy for Becker–– CANYON, the initial Phase 2 cohorts, is fully enrolled – BOULDER, Colo.–(BUSINESS WIRE)– Edgewise Therapeutics, Inc. (Nasdaq: […]
