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) […]
Dear Duchenne Community, We are pleased to announce that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on IGNITE DMD, our Phase I/II clinical trial. We […]
October 01, 2020 NEW YORK–(BUSINESS WIRE)– Pfizer Inc. (NYSE: PFE) today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track […]
–Results demonstrate continued safety and tolerability of SRP-9001 in four participants with Duchenne — –All four participants demonstrated improvements in NSAA scores compared to baseline and showed a durable response two years after administration of SRP-9001 — CAMBRIDGE, Mass., Sept. 28, 2020 (GLOBE NEWSWIRE) — Sarepta […]
