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 […]
Newport Beach, CA, December 21, 2020 – CureDuchenne, a leading global nonprofit focused on funding and finding a cure for Duchenne muscular dystrophy, announced today a new initiative to deliver […]
Dear Committee Members, On behalf of individuals living with Duchenne muscular dystrophy, their families, and caregivers served by CureDuchenne, I am writing to urge the Committee to include individuals with Duchenne in its recommendations for sub-populations to receive approved or authorized COVID-19 […]
Loss of bone density and strength is a common problem in Duchenne muscular dystrophy, and can lead to frequent fractures, chronic spine pain and deformity, and premature loss of ambulation. […]
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 […]
Debra Miller, Founder and CEO of CureDuchenne, and Dr. Peter Marks, Director of the Center for Biological Evaluation and Research of the FDA, were featured at a Fireside Chat at […]
