Henri Termeer joins Prosensa as Strategic Advisor
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 […]
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), […]
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), […]
New formulation delivers drug levels that are predicted to significantly increase utrophin production Summit to progress utrophin upregulator into next stages of development Oxford, UK, 7 November 2012 – Summit […]
A recent paper from Elisabeth Rumeur and colleagues (https://www.ojrd.com/content/pdf/1750-1172-7-45.pdf) introduces a new database (https://edystrophin.genouest.org/) that is made freely available for public access. It contains information from 945 clinical reports.
The eDystrophin database compliments two other databases of DMD human mutations: the Leiden Muscular Dystrophy database and the UMD-DMD French database. The eDystrophin database is specifically dedicated to providing information about in-frame mutations (deletions, duplications and substitutions) of the DMD gene and the consequences of these alterations from a clinical perspective and a protein structure perspective.
The database is user friendly and informative and should be a source of much needed information for researchers, parents and patients alike.
Myostatin is both a regulator of muscle growth and a stimulator of muscle fibroblasts to proliferate; and there is an accumulating body of evidence that demonstrates inhibition of myostatin/ActRIIB signaling can ameliorate the pathology and function of dystrophic muscle in preclinical models of Duchene muscular dystrophy.