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 […]
The Coronavirus Aid, Relief, and Economic Security (CARES) Act encourages charitable giving by increasing tax incentives for individuals and corporations. We want to make sure that anyone who supports us […]
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 […]
CureDuchenne is pleased to share the news that Edgewise Therapeutics is starting a Phase 1 clinical trial to assess the safety, tolerability, and appropriate dose of their experimental therapeutic aimed […]
– EDG-5506 is a novel, oral, small molecule drug candidate with the potential to promote muscle function in patients with muscular dystrophy – Boulder, Colo., (November 11, 2020) – Edgewise […]
CureDuchenne Ventures is happy to announce an investment in Mesentech, a company based in Vancouver, Canada, that has a novel therapeutic approach for improving bone density and strength. This investment […]
