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 […]
As an early investor in Dyne Therapeutics, CureDuchenne is pleased share that Dyne announced positive data from their Phase 1/2 trial of DYNE-251 in individuals with Duchenne amenable to skipping […]
Letter to the Duchenne Community – INSPIRE DUCHENNE update Dear Duchenne Community, We are writing to share an important update on INSPIRE DUCHENNE, our Phase 1/2 clinical trial of SGT-003 for […]
Avidity Biosciences, which received early funding from CureDuchenne, announced positive initial data from their EXPLORE44 Trial for individuals with Duchenne amenable to skipping exon 44. Their experimental exon-skipping agent, AOC […]
