Cart
×
nNOS: How Important is it in 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 […]
CureDuchenne – Clinical Trials – FDA
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 […]
Sarepta Therapeutics Enters Into Collaboration for the Development of Additional Exon-Skipping Product for Duchenne Muscular Dystrophy
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), […]
Sarepta Therapeutics Enters Into Collaboration for the Development of Additional Exon-Skipping Product for Duchenne Muscular Dystrophy
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), […]
REPEAT DOSING OF SMT C1100 FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY MEETS ENDPOINTS IN PHASE 1 CLINICAL TRIAL
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 […]
Assessment of the structural and functional impact of in-frame mutations of the DMD gene, using the tools included in the eDystrophin online database.
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.
Inhibiting myostatin reverses muscle fibrosis through apoptosis.
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.
Sphingosine-1-Phosphate Enhances Satellite Cell Activation in Dystrophic Muscles through a S1PR2/STAT3 Signaling Pathway
A recent publication from Dr. Julie Saba et al., (Children’s Hospital Oakland Research Institute, Oakland, California, reference https://www.plosone.org/article/info%3Adoi%2F10.1371%2Fjournal.pone.0037218) highlights the role played by the signaling lipid sphingosine-1-phosphate (S1P) in controlling the fate of satellite cells (muscle stem cells) in mdx mice.