— FDA grants Priority Review Status and sets regulatory action date for February 25, 2021 —— FDA has indicated it does not currently plan to hold an advisory committee meeting […]
CureDuchenne congratulates the incredible Dyne Therapeutics team, who recently announced closing $115M in equity financing to develop transformational therapies for patients with serious muscle diseases. We share this news in […]
CureDuchenne is thrilled to relay the news that NS Pharma has received FDA marketing authorization for Vitolarsen, an exon 53 skipping therapy. The entire Duchenne community is grateful for NS […]
CureDuchenne is very proud that Dr. Kevin Flanigan, at Nationwide Children’s Hospital, has treated two patients with gene therapy that carry the Duplication 2 mutation. CureDuchenne funded Dr. Flanigan’s very […]
CAMBRIDGE, Mass., July 24, 2020 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has […]
This month’s Catabasis Connection:
Take this Survey by July 19! CureDuchenne is supporting the EveryLife Foundation for Rare Diseases in their effort to conduct a survey of families living with rare diseases. This survey […]
NS Pharma Announces Launch of Patient Support HubPARAMUS, NJ: July 7, 2020 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. […]
– EU label update supports Translarna use in patients who became non-ambulatory while on therapy – SOUTH PLAINFIELD, N.J., June 29, 2020 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee […]
