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 […]
Dear Duchenne community, Since 2016, Santhera has been conducting one of the largest clinical trials in Duchenne muscular dystrophy – the SIDEROS study. SIDEROS is a phase III double-blind, randomized, […]
This month’s Catabasis Connection:
Dear U.S. Duchenne community, As you know from our previous updates, the SIDEROS clinical trial is fully enrolled and Santhera has been working with the Food and Drug Administration (FDA) […]
