– 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 […]
– 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 […]
November 9, 2020 Results from all cohorts of PRECISION-HD1 and PRECISION-HD2 clinical trials and initial OLE data on track for 1Q 2021Dosing in three new clinical trials with novel compounds […]
11/05/20 4:02 PM EST — Dosing with SRP-9001 commercial-process material to proceed following discussions with U.S. FDA — — Clinical dosing is expected to begin before the end of 2020 — CAMBRIDGE, Mass., Nov. 05, 2020 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in […]
-Collaboration combines Solid’s differentiated microdystrophin construct and Ultragenyx’s HeLa PCL manufacturing platform for use with AAV8 and variants- -Solid receives $40 million upfront via equity investment at a premium; up […]
