To turn away from any member of the global Duchenne community would be to turn away from our own here at home. That’s why we at CureDuchenne, the non-profit […]
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review the New Drug Application (NDA) for drisapersen. The FDA is currently reviewing drisapersen for the treatment of patients with Duchenne muscular dystrophy amenable to exon 51 skipping. The advisory committee will review drisapersen data included in a new drug application (NDA) during a meeting on November 24, 2015.
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced results from the Phase 3, double-blind, placebo-controlled, 48-week ACT DMD trial of Translarnaâ„¢ (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The trial results showed clinically meaningful benefits for Translarna-treated patients.
After waiting for a very long time, the Duchenne community now has clarity on the Advisory Committee meetings dates for BioMarin and Sarepta. BioMarin’s date is November 24, 2015 […]
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) is tentatively scheduled to review Sarepta’s New Drug Application (NDA) for eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, on January 22, 2016.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced additional clinical efficacy and safety data from the Company’s Phase IIb program of eteplirsen in patients with Duchenne muscular dystrophy (DMD).
As a mom of a son with Duchenne, I understand the importance of physical therapy in this disease. Early after Hawken’s diagnosis, we were referred to a physical therapy […]
We are delighted to announce that Katie Mastro has joined joined CureDuchenne as program services manager. She is responsible for managing and expanding the CureDuchenne Cares program, engaging with […]
The CureDuchenne Cares Certification Program will train physical therapists on the most current standards of care for Duchenne through a continuing education program including ongoing training and testing. The program […]
