-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 […]
Pratteln, Switzerland, September 22, 2020 – Santhera Pharmaceuticals (SIX: SANN) announces that partner ReveraGen Biopharma Inc. and their academic collaborators have published new open-label, long-term clinical data on the safety, tolerability […]
CureDuchenne celebrates Dyne Therapeutics’ public offering on the Nasdaq, under the ticker “DYN”. Today’s initial public offering (IPO) will elevate Dyne’s financial position and accelerate Dyne’s lead programs, which includes […]
