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 […]
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 […]
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 […]
The del-zota Managed Access Program (MAP) is now live on clinicaltrials.gov: 11/26/2025 Letter to the community added: 11/20/2025 Avidity Managed Access Program As an early funder of Avidity Biosciences, CureDuchenne is so happy […]
As an early investor in Entrada Therapeutics, we are pleased to share their progress on their exon-skipping programs in Duchenne. For Exon 44: Entrada completed enrollment for the first cohort […]
Capricor Therapeutics, which received early funding from CureDuchenne, provided an update on their Deramiocel program in Duchenne after their recent FDA meeting. This meeting with the FDA was to address […]
As an early investor in Avidity Biosciences, we are pleased to share their exciting news that skipping exon 44 in the dystrophin gene with Del-zota was associated with functional improvements […]
