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. […]
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. […]
Solid Biosciences reports that 33 children have now been dosed in their SGT-003 gene therapy trial for Duchenne. So far, the treatment has been generally well tolerated with signs of improved muscle […]
Capricor Therapeutics, which received early funding from CureDuchenne, said the FDA has requested the full clinical study report from their latest clinical trial, the HOPE-3 trial. Positive topline results from […]
World Duchenne Day 2025
REGENXBIO announced new, positive 18-month functional data from patients treated with the pivotal dose in the Phase I/II portion of the AFFINITY DUCHENNE® trial. They expect to share pivotal topline data […]
As an early investor in Entrada Therapeutics, we are pleased to share that in 2026 Entrada plans to have four clinical-stage programs in Duchenne – for individuals amenable to skipping […]
CureDuchenne applauds the U.S. Department of Health and Human Services (HHS) for adding Duchenne muscular dystrophy to the Recommended Uniform Screening Panel (RUSP) for newborn screening. We also extend our congratulations to Parent Project […]
Satellos Biosciences has received Investigational New Drug (IND) clearance from the FDA, as well as other global regulatory agencies, to conduct a 3-month placebo-controlled Phase 2 study of SAT-3247 in […]
