Muscular Dystrophies

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A small-molecule compound counteracts some of the effects of abnormal genetic instructions in MMD1

posted on November 3, 2009 - 10:00pm
  A compound that has the potential to be refined and modified into a treatment for type 1 myotonic dystrophy (MMD1, or DM1) has been identified by researchers at the University of Oregon in Eugene, and the University of Rochester (N.Y.) School of Medicine and Dentistry.
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Injections of genes for the muscle-growth protein follistatin strengthened leg muscles in monkeys

posted on November 12, 2009 - 10:00pm
Four macaque monkeys that received injections of genes for a protein called follistatin into upper leg muscles experienced pronounced and durable increases in muscle size and strength with no adverse effects, say researchers at Nationwide Children's Hospital in Columbus, Ohio, and Ohio State University. The findings could have implications for people with muscular dystrophies and other...
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A compound that seals cell membranes stopped progression of heart degeneration in dogs with a DMD-like disease

posted on March 22, 2010 - 2:19pm
A synthetic compound that seals cellular membranes has been found to stop the progression of heart-muscle destruction in dogs with a disease closely resembling human Duchenne muscular dystrophy (DMD), MDA-supported researchers have found.
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"This Month in Muscular Dystrophy" is the subject of a new audio series from Nationwide Children's Hospital in Columbus, Ohio

posted on May 24, 2010 - 3:53pm
Scientists at the Neuromuscular Disorders Program at Nationwide Children’s Hospital in Columbus, Ohio, are producing a series of podcasts examining current research in neuromuscular disease. The series, “This Month in Muscular Dystrophy,” is an opportunity to hear authors of recent publications discuss how their work improves understanding of these diseases, and what it might mean for treatment.
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Researchers report making a 10-fold improvement in a technology that permanently repairs flawed dystrophin genes, giving a real boost to this therapeutic strategy

posted on June 29, 2010 - 9:40am
A new generation of molecules — peptide nucleic acid single-stranded oligodeoxynucleotides or PNA-ssODNs — can help cells permanently repair errors in the dystrophin gene, fixing the underlying cause of Duchenne muscular dystrophy (DMD), researchers report. The research group, headed by MDA grantee, Carmen Bertoni, at the University of California Los Angeles (UCLA), published its findings online...
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Sidelined by bone cancer, limb-girdle MD and depression, Steve Williams found a way to get back out on the open road

posted on July 20, 2010 - 3:55pm
The “passenger” in Steve Williams’ motorcycle sidecar doesn’t say much, but if it could talk, it probably would have plenty of stories to tell.
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Brief items about myostatin inhibition, exon skipping and stem cell research in Duchenne muscular dystrophy

posted on October 20, 2010 - 9:45am
Anti-myostatin drug trial shows good preliminary results
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A new study seeks to investigate the possible relationship between religion/spirituality and disability acceptance

posted on November 10, 2010 - 5:01pm
With approximately 54 million Americans living with disabilities, exploring and understanding factors that might facilitate or hinder acceptance of one’s disability may be an important area of research. But what exactly is the relationship between religious/spiritual attitudes and acceptance or lack of acceptance of disability for people with neuromuscular disorders?
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A variant in the gene for the osteopontin protein has the potential to help predict disease course in Duchenne MD

posted on January 11, 2011 - 2:58pm
A team of scientists working in the United States and Italy has uncovered a variant in the gene for a protein called osteopontin that appears to reliably indicate disease severity in most (but likely not all) cases of Duchenne muscular dystrophy (DMD). The variant is apparently a genetic modifier of DMD, a disease in which the underlying cause is a mutation in the gene for the dystrophin protein...
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Biologist Denis Guttridge discusses his team's work developing an NF-kappa B blocker as a potential treatment for Duchenne MD

posted on December 14, 2011 - 3:56pm
A December 2011 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores how an inhibitor of a protein called NF-kappa B has been beneficial in a mouse model of Duchenne muscular dystrophy (DMD) and is now being developed as a potential DMD treatment. The podcast is part of a Nationwide Children's series called "This Month in Muscular Dystrophy."
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