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Catabasis Connection – July 2020
This month’s Catabasis Connection:
Embrace This Moment – Part 3
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 […]
EveryLife Survey – What’s the cost of rare disease to YOU?
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 Hub
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. […]
Embrace This Moment – Part 2
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 […]
PTC Therapeutics Announces CHMP Recommendation of Translarna™ (ataluren) Label Update for Non-Ambulatory Patients with Duchenne Muscular Dystrophy
– 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 […]
Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045) for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45
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 […]
Sarepta Pipeline Presentation
In this webinar, Sarepta Therapeutics presents an update on their Duchenne muscular dystrophy pipeline. Diane Berry, PhD, Senior Vice President, Global Health Policy, Government and Patient Affairs and Louise Rodino-Klapac, […]
CureDuchenne Webinar: An Introduction to NS Pharma, Inc. and its access support program, NS Support
During this webinar, you will learn about NS Pharma, Inc. (a wholly owned, US subsidiary of Nippon Shinyaku Co., Ltd. based in Kyoto, Japan), focused on developing innovative treatments for […]