CureDuchenne was honored to host a webinar today, in collaboration with PPMD and MDA to discuss accelerated approval for drugs for rare diseases. Dr. Robert Temple, director of the […]
Leiden, The Netherlands – 29 January 2013 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, announced it has achieved orphan […]
GSK has sent out this update on their Duchenne trial. The last patient has been recruited within the drisapersen US Phase II clinical study [www.clinicaltrials.gov NCT01462292]. This exploratory study aims […]
Exon duplication mutations have been an area of great interest to CureDuchenne, and last year, CureDuchenne funded research specifically directed toward these mutations in Duchenne. Dr. Kevin Flanigan at Nationwide […]
We are so pleased to see the high caliber of professionals joining in the fight to cure Duchenne. Henri Termeer served as Chairman, President and CEO of Genzyme Corporation for […]
Drs. Carrie Miceli and Stan Nelson at UCLA are developing an FDA approved drug that could boost the effectiveness of exon skipping drugs that are being developed and tested for Duchenne […]
nNOS has been in the news lately so we asked CureDuchenne’s chief scientific advisor, Dr. Mike Kelly to answer a few questions. Question: what is nNOS and why do we need it? nNOS […]
Following the long awaited news of Sarepta’s phase IIB results for eteplirsen and continued Phase III progress of GSK/Prosensa’s drisapersen, as well as the recent developments with PTC Therapeutics and […]
November 26, 2012 08:30 ET Collaboration Represents Significant Progress in Sarepta’s Path to Develop Treatments for Broader DMD Population CAMBRIDGE, MA–(Marketwire – Nov 26, 2012) – Sarepta Therapeutics (NASDAQ: SRPT), […]
