Summary from Seeking Alpha Based on updated guidance from the FDA regarding an  early approval pathway for eteplirsen, Sarepta Therapeutics (SRPT) plans to file an NDA by the end of 2014. The agency provided specific examples of additional  safety and efficacy data for Duchenne muscular dystrophy that would  enhance the acceptability of the NDA. The company will conduct […]
Congratulations to the Race to Yes organizers (Tracy Seckler, Jenn McNary, Christine McSherry, Mindy Leffler, and many others) and the Duchenne community for their passionate and successful campaign to petition the White House to urge the Food and Drug Administration (FDA) to use  the Accelerated Approval pathway for approval and access to safe, effective therapies […]
Prosensa will present a status update on Drisapersen and its follow-on compounds in a patient focused webinar hosted by United Parent Projects Muscular Dystrophy (UPPMD) on Tuesday, March 25 at 1 p.m. ET. If you have any questions regarding recent Prosensa/drisapersen updates or any other topic related to the company’s Duchenne portfolio that you would […]
        Clinically meaningful improvement from 24-week treatment period was maintained for 24 weeks after drisapersen administration ceased Chicago, IL, March 17, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today reported encouraging 48-week  data from its […]
CureDuchenne will host our annual Champions to CureDuchenne Newport Beach Gala on Saturday, March 29 at the Balboa Bay Club & Resort. The event will celebrate CureDuchenne’s 10 years of successfully advancing Duchenne research. We’ll honor Doug Freeman, the business leader who helped shape our venture philanthropy model; dine on a delicious meal created by […]
Leiden, February 18th , 2014 Dear Patient Group Representative, We hope this note finds you well. When we last communicated, we announced that Prosensa had regained all rights to drisapersen from GSK and retained the rights to the other DMD programs. Upon the announcement of the transfer of rights, GSK and Prosensa entered into a […]

Prosensa Patient Webinar

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Dr. Giles Campion, Prosensa’s Chief Medical Officer (http://www.prosensa.eu/) presented an overview of the latest drisapersen results in this morning’s patient webinar. An analysis of the Phase III data suggests that administering the drug earlier in the disease and treating longer can delay the progression of DMD. For example, in the 96-week extension data from the […]
Dear all, As announced last week, Prosensa will organize a patient focused webinar on Tuesday 21st of January at 8 AM ET / 14hrs CET. Practical details required to dial in for this webinar are provided on the Prosensa website at:  http://ir.prosensa.eu/events.cfm (open additional information by clicking on it). If you have any questions that […]
Leiden,The Netherlands,Jan. 16, 2014(GLOBE NEWSWIRE) –Prosensa Holding N.V.(NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet medical need, today announced initial findings from further analyses from the aggregate data from the clinical development program of drisapersen for the treatment of Duchenne Muscular Dystrophy (DMD). “We are encouraged by […]
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jan. 15, 2014– Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced data through Week 120 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results through more than two years showed a continued stabilization of walking ability in eteplirsen-treated patients evaluable […]
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