While there is currently no cure for Duchenne muscular dystrophy, gene therapy has the potential to target the underlying genetic cause of many mono genetic diseases and could potentially benefit […]
The positive results from the 12-month ARCH study support the hypothesis that a reduction in contraction-induced muscle damage in muscular dystrophies, associated with EDG-5506 administration, has the potential to preserve and improve muscle function while preventing disease progression in dystrophinopathies. Observations from ARCH identified key factors, including the optimal dosing strategy of EDG-5506, for the design of a potentially registrational trial. A pivotal cohort, GRAND CANYON, has been added to the CANYON study and is anticipated to begin enrollment in the third quarter of 2023.
SEE UPDATE FROM OCTOBER 30, 2023 HERE CLICK BELOW FOR A FEW WORDS FROM OUR TEAM AND SAREPTA: Sarepta Therapeutics’ gene therapy for Duchenne, SRP-9001, has been granted Accelerated Approval […]
My name is Debra Miller, founder and CEO of CureDuchenne, and the mother of an incredible son, Hawken, who has Duchenne muscular dystrophy. I’m here today to represent the voice of patient advocacy organizations serving those impacted by Duchenne – all of whom are in support of the accelerated approval of SRP-9001.
See the below letter to the community
Join us for this pre-recorded presentation and Q&A discussing Dyne’s Phase 1/2 clinical trial, DELIVER, evaluating DYNE-251 for the treatment of individuals with Duchenne muscular dystrophy (DMD) who are amenable […]
PARAMUS, NJ: April 14, 2023,–NS Pharma, Inc. announced today the U.S. Food & Drug Administration (FDA)has agreed to the planned Phase II study of NS-089/NCNP-02 for Duchenne muscular dystrophy. NS-089/NCNP-02 […]
Dyne Therapeutics, who received early funding from CureDuchenne, has received FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy.
CureDuchenne was the original funder of cTAP. We congratulate the consortium and other authors on their recent publication looking at large data sources and investigating effects of different genotypes on […]
