As an early funder of Edgewise, CureDuchenne is pleased to share that the Phase 2 Trial of sevasemten in Becker muscular dystrophy met its primary endpoint of reduction in creatine […]
CureDuchenne is delighted to share the positive updates from REGENXBIO on their Phase 1/2 open-label clinical trial of RGX-202, an AAV8-delivered microdystrophin with an extended C-terminal domain. RGX-202 demonstrated robust […]
Updates from the INSPIRE DUCHENNE clinical trial of SGT-003, SolidBio’s investigational, next-generation AAV microdystrophin gene therapy candidate brings expanded eligibility criteria, additional trial sites in more regions, and upcoming preliminary data. Both the […]
CureDuchenne is pleased to share that PTC Therapeutics has announced that the FDA has accepted for review the New Drug Application (NDA) for Translarna (ataluren) for the treatment of Duchenne […]
Watch the video HERE Pfizer recently provided this video updating the results from their Phase 3 micro-dystrophin gene therapy trial, CIFFREO, which were presented on October 12, 2024 at the […]
CureDuchenne Shares Updates from 29th Annual Congress of the World Muscle Society We’re at the 29th Annual Congress of the World Muscle Society this week and are pleased to share […]
As an early investor in Capricor Therapeutics, CureDuchenne is happy to share that Capricor has announced their intent to apply to the FDA for full approval of Deramiocel (CAP-1002) for […]
CureDuchenne is pleased to communicate that Wave Life Sciences announced positive interim data from the ongoing Phase 2 FORWARD-53 study in Duchenne amenable skipping exon 53. After 24 weeks of […]
SEE THE FULL PRESS RELEASE BELOW AND HERE – Second annual DREAMS Grant Program awards $25,000 each to three U.S.-based non-profit organizations working to achieve greater equality for those living […]
