Articles
Sarepta Therapeutics Announces Significant Clinical Benefit With Eteplirsen After 36 Weeks in Phase IIb Study for the Treatment of Duchenne Muscular Dystrophy
July 24, 2012Sarepta Therapeutics (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced that treatment with its exon-skipping compound, eteplirsen, achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT), over a placebo/delayed treatment cohort in a Phase IIb trial in Duchenne muscular dystrophy (DMD) patients.
Read moreAssessment of the structural and functional impact of in-frame mutations of the DMD gene, using the tools included in the eDystrophin online database.
July 9, 2012A recent paper from Elisabeth Rumeur and colleagues (http://www.ojrd.com/content/pdf/1750-1172-7-45.pdf) introduces a new database (http://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.
Read moreNon-invasive prenatal measurement of the fetal genome
July 4, 2012Researchers at Stanford University have for the first time sequenced the genome of an unborn baby using only a blood sample from the mother.
Read moreInhibiting myostatin reverses muscle fibrosis through apoptosis.
June 8, 2012Myostatin 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.
Read moreSphingosine-1-Phosphate Enhances Satellite Cell Activation in Dystrophic Muscles through a S1PR2/STAT3 Signaling Pathway
May 14, 2012A recent publication from Dr. Julie Saba et al., (Children's Hospital Oakland Research Institute, Oakland, California, reference http://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.
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