Research News
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May 17, 2013
Interim results from a phase 1 clinical trial of RG2833 in people with Friedreich’s ataxia (FA) show that the experimental drug is well-tolerated, and that it appears to increase the activity of the
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May 17, 2013
The Discretionary Advisory Committee on Heritable Disorders in Newborns and Children (DACHDNC) today voted to add Pompe disease (acid maltase deficiency) to a list of diseases that it recommends
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May 16, 2013
A phase 2 clinical trial to test the experimental drug GM604 in people with amyotrophic lateral sclerosis (ALS) has opened at two trial sites in the United States. Enrollment is expected to begin
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May 10, 2013
Melatonin, a naturally occurring hormone produced by the pineal gland, is best known for its role in regulating sleep. Now, results from a study supported in part by MDA show that treatment with the
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May 08, 2013
A large-scale, multinational phase 3 trial of the experimental drug ataluren has opened its first trial site, in Cincinnati, Ohio.
The trial is recruiting boys with Duchenne muscular dystrophy (DMD)
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May 07, 2013
A phase 2 clinical trial to test multiple doses of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA) has opened at four trial sites in the United States and Canada.
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May 03, 2013
The Myotubular Myopathy Event Study, a telephone-based survey, will gather information about MTM-associated events, such as emergency room visits, hospitalizations, medication reactions, and
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May 02, 2013
Biopharmaceutical companies Acceleron Pharma and Shire announced April 21, 2011, that MDA-supported clinical trials of ACE-031 for Duchenne muscular dystrophy (DMD) have been halted.
A phase 2, dose-
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April 25, 2013
Turning neuromuscular disease research into treatments as quickly and effectively as possible was the overarching theme of dozens of formal presentations, nearly 200 scientific posters, and countless
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April 24, 2013
As the conference approached the halfway point yesterday afternoon, between talks my mind occasionally wandered to nonscientific topics, like remembering to complete the online check-in for my
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April 24, 2013
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April 23, 2013
An MDA-supported research team has identified a series of compounds that appear to work both alone and in combination to protect the muscle-controlling nerve cells (motor neurons) that are lost in
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April 18, 2013
Cure CMD, in collaboration with the Congenital Muscle Disease International Registry (CMDIR), Valerion Therapeutics (formerly 4s3 Bioscience), the University of Chicago, University of Michigan and
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April 18, 2013
The Muscular Dystrophy Association’s annual conference being held in Washington, D.C., on April 21-24, 2013, is centered on the theme Therapy Development for Neuromuscular Diseases: Translating Hope
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April 17, 2013
MDA and the nonprofit biotech ALS Therapy Development Institute (ALS TDI) have extended their strategic research partnership through 2013. With the extension comes a $3.2 million MDA grant to help
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April 17, 2013
Maryland biotherapeutics company Neuralstem today announced that it has received approval from the U.S. Food and Drug Administration (FDA) to conduct a phase 2 clinical trial to test its NSI-566
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April 16, 2013
MDA has awarded $1 million to biopharmaceutical company ARMGO Pharma for development of a new strategy for treating Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD).
The
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April 15, 2013
The U.S. Food and Drug Administration (FDA) has said it will consider an application for accelerated approval for eteplirsen, an experimental drug for Duchenne muscular dystrophy (DMD), after it
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April 15, 2013
In the search for therapies for spinal muscular atrophy (SMA), researchers are testing two drugs with connections to other neuromuscular disorders: riluzole, which is approved for use in amyotrophic
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April 11, 2013
The presence of cognitive impairment within the first year after a diagnosis of amyotrophic lateral sclerosis (ALS) may be associated with more rapid decline in muscle function, a team of researchers
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April 11, 2013
The exon-skipping drug drisapersen has shown encouraging results in an international phase 2b trial in boys with Duchenne muscular dystrophy (DMD) caused by specific mutations.
After 48 weeks of
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April 05, 2013
Eteplirsen, an experimental exon-skipping therapy designed to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show sustained benefit on
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April 04, 2013
A newly developed research mouse that has the same combination of genetic alterations that causes human facioscapulohumeral muscular dystrophy (FSHD) is expected to change the way research in this
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