ITF shared a community letter and presented new data, including long-term safety observations based on data from the company’s ongoing open-label extension study in patients with Duchenne muscular dystrophy treated with […]
REGENXBIO GENE THERAPY RGX-202, an investigational gene therapy for Duchenne, continues to show a favorable safety profile with no serious adverse events, no liver injury signals, and reductions in key […]
Santhera Pharmaceuticals announced results from the ongoing Guardian study in Duchenne, and showed that AGAMREE had comparable effectiveness compared to historical cohorts taking prednisone or deflazacort, based on time to […]
As an early funder of Entrada Therapeutics, CureDuchenne is pleased to share that after the independent Data Monitoring Committee reviewed the safety data from Cohort 1 of the ELEVATE-44-201 trial […]
Solid Biosciences shared that they have aligned with the US FDA on the overall design of a Phase 3 randomized, double-blind, placebo-controlled trial for SGT-003, a micro-dystrophin gene therapy for Duchenne. […]
Sarepta Therapeutics announced positive topline data from the EMBARK trial, showing that Elevidys, 3 years post treatment, significantly slows disease progression on functional measures in ambulatory individuals who were 4-7 […]
Avidity Biosciences has released answers to frequently asked questions about their Managed Access Program (MAP) for individuals with Duchenne who are amenable to exon 44 skipping and meet clinical criteria. […]
World Duchenne Day 2025
A presentation with Dr. Doug Kerr, Chief Medical Officer at Dyne Therapeutics. Dr. Kerr shares topline results from the DELIVER clinical trial, along with new long-term data showing sustained benefits […]
