Research News
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February 04, 2011
The Muscular Dystrophy Association has awarded 44 grants totaling $13.5 million to support research efforts aimed at advancing understanding of disease processes and uncovering new strategies for
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February 04, 2011
MDA has awarded eight new grants totaling nearly $2.5 million to fund research projects focused on uncovering the causes of, and developing therapies for, ALS. The effective start date for the grants
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February 02, 2011
Isis Pharmaceuticals hosted an interactive webinar Wednesday, March 2, 2011, at 12 noon Eastern Standard Time, to help prospective participants understand its MDA-supported, phase 1 study of ISIS-
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January 28, 2011
Update (Jan. 23, 2013): As of early 2013, MDA's DMD Clinical Research Network includes these five sites: University of California, Davis (UC Davis); Nemours Children's Hospital in Orlando, Fla.;
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January 26, 2011
Researchers at the University of Michigan are seeking 30 young adults, ages 18-29, who have had symptoms of certain forms of muscular dystrophy or myopathy since birth, to complete an online survey
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January 25, 2011
Researchers conducting a clinical trial of the antibiotic ceftriaxone in amyotrophic lateral sclerosis (ALS) currently are recruiting participants at 57 locations across the United States and Canada
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January 18, 2011
Experts from around the world will gather Jan. 21-22, 2011, at an MDA-sponsored conference about the heart in Duchenne muscular dystrophy (DMD).
With improvements in the care of people with DMD, life
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January 18, 2011
A new study to determine the best "outcome measure" (measurable activity) with which to assess thigh-muscle (quadriceps) strength in men with Becker muscular dystrophy (BMD) is seeking participants.
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January 14, 2011
MDA has begun funding tests of the experimental drug ACE-031 in children with Duchenne muscular dystrophy (DMD). The drug is being developed by Acceleron Pharma, a Cambridge, Mass., biotechnology
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January 13, 2011
A January 2011 podcast from Nationwide Children's Hospital in Columbus, Ohio, probes a subject that's been on the minds of many researchers, doctors and families: autoimmunity (self-immunity) in
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January 11, 2011
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
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December 29, 2010
Editor's note 2/7/11: A link to the Tivorsan Pharmaceuticals website has been added.
Duchenne and Becker muscular dystrophies
Scientists have found that systemically injecting the human form of a
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December 29, 2010
Spirulina supplement seems to help ALS mice
A study of 15 mice with a genetic mutation that causes an ALS-like disease suggests that the nutritional supplement spirulina may have some protective
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December 27, 2010
Update (Jan. 23, 2013): As of early 2013, MDA's DMD Clinical Research Network includes these five sites: University of California, Davis (UC Davis); Nemours Children's Hospital in Orlando, Fla.;
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December 23, 2010
Since 1993 — when mutations in the RYR1 gene were first linked to central core disease (CCD)— researchers have been trying to figure out exactly how these mutations cause the disease and what can be
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December 22, 2010
A multinational study group, using cutting-edge "exome sequencing" technology, has uncovered five mutations in the valosin-containing protein (VCP) gene and implicated them as molecular causes of
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December 22, 2010
A multinational study group, using cutting-edge "exome sequencing" technology, has uncovered five mutations in the valosin-containing protein (VCP) gene and implicated them as molecular causes of
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December 21, 2010
If your child participated in a phase 2 clinical trial of ataluren (PTC124) for Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) conducted by PTC Therapeutics, you're invited to
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December 21, 2010
New insights and perspectives on the biology of amyotrophic lateral sclerosis (ALS), best practices in the care of individuals with the disease, and future directions for development of ALS therapies
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December 16, 2010
People with genetic neuromuscular diseases who want to “do something for science” now have a way to do so, although they’re unlikely to ever know the results of their good deed.
Scientists at the
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December 15, 2010
A large-scale, phase 3 trial of idebenone (Catena) in Duchenne muscular dystrophy (DMD) is now open at one U.S. site and several sites in Europe, under the auspices of Santhera Pharmaceuticals.
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December 15, 2010
MDA has awarded a $1.4 million grant to the biopharmaceutical company Repligen Corp. to help advance the company’s experimental drug for spinal muscular atrophy (SMA) to phase 1 human clinical trials
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December 10, 2010
Immune system regulator shows safety in ALS
Neuraltus Pharmaceuticals announced Nov. 30, 2010, that its experimental drug NP001 was safe and well-tolerated at four different dose levels when given as
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December 09, 2010
Dystrophin-deficient mice (known as mdx mice) have been used in experiments as a model of human Duchenne muscular dystrophy (DMD) for decades. However, scientists have long noted that mdx mice, even
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December 09, 2010
Duchenne and Becker muscular dystrophies
PTC Therapeutics says it will provide its experimental drug ataluren to those who participated in its ataluren trials conducted in the United States. Ataluren
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