Research News
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February 06, 2013
A 12-participant study of the acute effects of two vasodilating drugs on blood flow to exercising muscles needs four more boys with Duchenne muscular dystrophy (DMD) who meet study criteria and are
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February 04, 2013
In August 2012, MDA awarded a research grant totaling $301,614 over three years to Marc Weisskopf; the funds are helping support Weisskopf's work to identify nongenetic risk factors for amyotrophic
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February 04, 2013
Twelve new grants totaling $3.6 million have been awarded in support of research studies that will explore the causes of, and potential treatments for, amyotrophic lateral sclerosis (ALS).
“This is
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February 04, 2013
Everyone is born, and everyone dies. What happens in between — colds and flu, cardiac problems, cancer, amyotrophic lateral sclerosis (ALS) — is anyone's guess. And people do guess. It's common —
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February 04, 2013
The identification of nongenetic risk factors can help scientists better understand and determine the causes of ALS. The work to uncover these factors is being facilitated by the National ALS
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February 03, 2013
A recently opened global registry— a database of patient information — is seeking people with the type 2A form of limb-girdle muscular dystrophy (LGMD2A), a form of LGMD that results from a
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January 25, 2013
Development of stem cell therapy to treat amyotrophic lateral sclerosis (ALS) continued with two studies in mice, one by an Israeli biotech company and one by an Italian research team.
Repeat doses
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January 23, 2013
A survey of people with inclusion-body myositis (IBM) is being conducted by A. David Paltiel, a professor of public health (health policy) and management at Yale University, with colleagues there and
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January 23, 2013
MDA has launched a new, five-center clinical research network focused on type 1 and type 2myotonic muscular dystrophy (MMD1 and MMD2, also known as DM1 and DM2), with the principal goal of preparing
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January 20, 2013
Mice with a disorder resembling the type 1B form of Charcot-Marie-Tooth disease (CMT) benefited from treatment with either of two forms of oral curcumin, a component of the spice turmeric, according
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January 15, 2013
Edison Pharmaceuticals has launched a phase 2b clinical trial of its experimental drug EPI-743 in adults with Friedreich's ataxia (FA) to assess whether the drug has positive effects on visual
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January 15, 2013
For the first time, gene therapy using a highly miniaturized dystrophin gene resulted in significant improvement in muscle structure and function in dogs with a disorder mimicking human Duchenne
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January 15, 2013
In early January 2013, Biogen Idec announced that, unfortunately, its phase 3 "EMPOWER" trial of dexpramipexole showed that the drug had no apparent effect on function or survival in amyotrophic
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January 10, 2013
Mice missing the muscle protein myotubularin and showing a disorder mimicking human myotubular myopathy (MTM) showed improvements in muscle structure and function after two weeks of treatment with a
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January 09, 2013
The Israeli Ministry of Health has given BrainStorm Cell Therapeutics approval to accelerate its current phase 1-2 safety trial of the company’s NurOwn stem cell therapy in amyotrophic lateral
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January 07, 2013
MDA is pushing forward on all levels to accelerate the pace at which we’re able to translate scientific discoveries into improved health outcomes for individuals living with muscle disease.
Longtime
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January 07, 2013
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ALS (amyotrophic lateral sclerosis)
Regulatory Cells Associated with Speed of ALS Progression, November 26
ALS
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January 04, 2013
The biotechnology firm Repligen today announced it has entered into an agreement with the global pharmaceutical company Pfizer to advance Repligen’s spinal muscular atrophy (SMA) program, which
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January 02, 2013
Biotechnology company Biogen Idec today announced disappointing results from its phase 3 trial of dexpramipexole, an experimental drug the company has been developing to treat amyotrophic lateral
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December 31, 2012
In a December 2012 podcast from Nationwide Children's Hospital, cell biologist Federica Montanaro discusses her team's recent progress in understanding how various proteins interact with dystrophin
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December 27, 2012
Below is a wrap-up of recent research news about the development of therapies for Duchenne, Becker and limb-girdle muscular dystrophies.
Follistatin compound gains orphan drug status
A potentially
December 26, 2012
Update (Jan. 23, 2013):The "Building better utrophin" section was updated to reflect the availability of a Jan. 22, 2013, press release from the University of Missouri.
Below is a wrap-up of recent
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December 24, 2012
A small-molecule drug candidate for spinal muscular atrophy (SMA) and an effective strategy for advancing therapy development are the primary outcomes being reported by the Spinal Muscular Atrophy (
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December 20, 2012
Injecting a muscle-repair protein known as WNT7a into the muscles of mice lacking the dystrophin protein and showing a disease resembling Duchenne muscular dystrophy (DMD) significantly increased
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