PTC Therapeutics shared the below letter to the Duchenne community. We are pleased to see the results of the dystrophin study (Study 045) and thankful for PTC’s longtime commitment to […]
CureDuchenne is pleased to share news that Avidity Biosciences has announced exciting details of its Duchenne program today, the full press release is below. CureDuchenne Ventures made an investment in […]
— Study met the primary biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment, as measured by western blot, in SRP-9001-treated participants versus placebo — — SRP-9001-treated participants showed […]
NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE: PFE) today announced that the first participant has been dosed in the Phase 3 CIFFREO study, which will evaluate the efficacy and safety of investigational […]
– Pivotal program for RGX-314, potential best-in-class, one-time gene therapy for the treatment of wet AMD, is active and expected to support BLA filing in 2024— Recently completed an End […]
CureDuchenne Ventures is happy to announce its latest research funding is an investment in PepGen, a therapeutics company targeting Duchenne muscular dystrophy and other neuromuscular diseases with next-generation antisense oligonucleotides. […]
— Results from the multiple–ascending dose trial demonstrate proof-of-concept for SRP-5051 and support continued dose escalation — — At a total dose exposure approximately 10x lower than eteplirsen, SRP-5051 at 20 mgs/kg showed enhanced tissue exposure, greater […]
CAMBRIDGE, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Monday, Dec. 7, 2020 at […]
In October 2020, Santhera Pharmaceuticals announced the disappointing news that it was discontinuing its Phase 3 SIDEROS trial of Puldysa (idebenone) in Duchenne muscular dystrophy. The data from a pre-planned […]
