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. […]
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 […]
We congratulate C-PATH (Critical Path Institute) on their success in getting the FDA to qualify a new biomarker– glutamate dehydrogenase (GLDH)– to monitor drug-induced liver injury in clinical trials. This […]
Letter to the community added November 17, 2025 The FDA has approved the new labeling information Sarepta provided for Elevidys, Sarepta’s gene therapy for Duchenne. This update comes after reports […]
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 […]
