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:
Part 3 of the series engages adults with Duchenne and families who have been on the Duchenne journey for more than a few years, or those who could benefit from […]
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. […]
Part 2 of the series engages families who have carved out their path on the Duchenne journey for a few years, or those who have foundational knowledge of Duchenne and […]
– 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 […]
06/26/20 8:30 AM EDT — Casimersen is designed for the treatment of exon 45 amenable patients, approximately eight percent of patients with Duchenne — — Casimersen is the third exon-skipping […]
