Research News
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December 18, 2012
The 23rd International Symposium on ALS/MND (motor neuron disease), held in Chicago Dec. 5-7, 2012, brought together more than 900 researchers, clinicians and other health care professionals from 30
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December 17, 2012
Scientists supported in part by MDA have developed a second type of research mouse with a disorder mimicking hypokalemic periodic paralysis, a genetic disorder in which recurrent attacks of weakness
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December 13, 2012
The 23rd International Symposium on ALS/MND (motor neuron disease), held in Chicago Dec. 5-7, 2012, brought together more than 900 researchers, clinicians and other health care professionals from 30
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December 12, 2012
The experimental drug tirasemtiv has shown promise in a phase 2a clinical trial in 32 people with myasthenia gravis (MG). Improvements in general and respiratory muscle function occurred in response
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December 07, 2012
Eteplirsen, an exon-skipping drug in development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show benefit at
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December 07, 2012
In both October and December 2012, Sarepta Therapeutics announced very encouraging results from a 12-person phase 2b trial of eteplirsen, an exon-skipping compound that is a potential treatment for
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December 05, 2012
New Jersey biopharmaceutical company PTC Therapeutics has announced that the European Medicines Agency (EMA) has validated a "marketing authorization application" (MAA) seeking conditional approval
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December 04, 2012
Protein test predicts ALS progression
It’s known that levels of a protein called phosphorylated neurofilament heavy subunit (PNF-H) are increased in the spinal fluid of people with amyotrophic
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December 04, 2012
Biopharmaceutical company Sarepta Therapeutics has announced it will expand the focus of its exon-skipping program for Duchenne muscular dystrophy (DMD) by developing compounds that target exons 45,
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November 28, 2012
The drug tadalafil (Cialis, Adcirca), which dilates blood vessels and is approved to treat erectile dysfunction and pulmonary hypertension, has been found to improve blood flow to exercising forearm
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November 26, 2012
Update (Dec. 20, 2012):This story has been updated to reflect the availability of a 12-minute podcast with researcher Michael Rudnicki, in which Rudnicki discusses the advantages and disadvantages of
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November 25, 2012
Immune system cells known as regulatory T cells— T-regs for short — have been found to play a protective role in mice with a disorder that resembles human amyotrophic lateral sclerosis (ALS). They
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November 19, 2012
A unique international contest organized by Boston Children’s Hospital has solved a genetic mystery for the family of a boy diagnosed with centronuclear myopathy (CNM).
From left: A 2007 photo shows
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November 18, 2012
Findings from a study of 516 Italian boys show a significant improvement over the last six decades in survival time in Duchenne muscular dystrophy (DMD). This modified natural history (general
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November 15, 2012
Edison Pharmaceuticals has announced it is conducting a clinical trial of its experimental drug EPI-743 in children with the mitochondrial myopathyLeigh syndrome, and is planning a trial of this
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November 15, 2012
A phase 1b/2a trial to test the safety and tolerability of multiple doses of the experimental drug ISIS-SMNRx in children with spinal muscular atrophy (SMA) is now open at sites in New York and Salt
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November 13, 2012
Nerve cells called serotonergic neurons degenerate in amyotrophic lateral sclerosis (ALS) and may influence the ALS disease process, an international research team has reported. In particular, the
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November 11, 2012
Scientists funded in part by MDA have found the cause of type 2facioscapulohumeral muscular dystrophy (FSHD), a discovery that increases scientific understanding of both forms of FSHD, will improve
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November 11, 2012
A webinar (Web-based seminar) about neurodegenerative causes of death, including amyotrophic lateral sclerosis (ALS), in retired National Football League (NFL) football players is scheduled for 1 p.m
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November 04, 2012
Results from a completed phase 2 trial of the experimental therapy NP001 in people with amyotrophic lateral sclerosis (ALS) show that in some trial participants, the drug appeared to be effective at
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November 02, 2012
The multinational pharmaceutical company GlaxoSmithKline (GSK) has announced promising results for its phase 1 trial of the exon-skipping drug drisapersen in boys with Duchenne muscular dystrophy (
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November 01, 2012
November 01, 2012
The ALS Association and Muscular Dystrophy Association (MDA) appreciate the opportunity to comment on the Patient-Focused Drug Development Initiative and the Agency’s preliminary list of nominated
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October 28, 2012
Compounds targeting exon 52 and exon 55 of the dystrophin gene as a way to treat some forms of Duchenne muscular dystrophy (DMD) will be moved into clinical trials as soon as possible, says Dutch
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October 25, 2012
It's been known since the early 1990s that mutations in the superoxide dismutase-1 (SOD1) gene can result in the production of any number of varieties of improperly folded (misfolded) SOD1 protein,
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