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 […]
As an early investor in Dyne Therapeutics, we are pleased to share their exciting news that skipping exon 51 in the dystrophin gene with z-rostudirsen in the registrational expansion cohort […]
Capricor, which received early funding from CureDuchenne, announced today that HOPE-3 met its primary endpoint and a key cardiac secondary endpoint, with all Type 1 error–controlled secondary endpoints also achieving […]
Wave Life Sciences has announced that they are initiating Part C of the FORWARD-53 trial—an open-label Phase 1b/2 study of up to 15 ambulatory individuals aged 4-10 years and amenable […]
Sarepta Therapeutics has announced that the FDA has approved Cohort 8 of the ENDEAVOR study, to evaluate the use of immunosuppression with sirolimus as part of treatment with Elevidys in […]
In 2018, CureDuchenne was the only Duchenne-focused organization to invest in Avidity Biosciences, recognizing the potential of their novel RNA-targeting approach for individuals with Duchenne muscular dystrophy long before it reached clinical trials. […]
