Research News
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July 24, 2012
Editor's note (July 24, 2012): For a more in-depth discussion of the exon-skipping trial results, see A Closer Look: Extended Eteplirsen Treatment Benefits Walking in DMD.
The biopharmaceutical
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July 18, 2012
Update (Aug. 8, 2012):This story was updated to reflect the availability of a podcast on the dysferlin gene transfer study.
Zebrafish research models mimic myofibrillar myopathy
Scientists in
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July 15, 2012
Disruption of a “transporter” protein called MCT1 (also SL16A1) leads to the degeneration of muscle-controlling nerve cells (motor neurons) in animal and cell culture models of amyotrophic lateral
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July 10, 2012
Type 1 myotonic dystrophy (MMD1, or DM1) and type 2 myotonic dystrophy (MMD2, or DM2) are complex, multisystem disorders caused by similar genetic flaws on chromosome 19 (MMD1) and chromosome 3 (MMD2
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July 09, 2012
In a more than 20-year study of people with amyotrophic lateral sclerosis (ALS), a research team found that 11 percent of people with a diagnosis of sporadic ALS had mutations in genes associated
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July 04, 2012
New research mouse mimics McArdle disease
Scientists in Spain have developed mice with a disorder that closely resembles the human metabolic muscle disorder known as McArdle disease (also called
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July 03, 2012
LGMD2D mice benefit from corrected human stem cells
A multinational team of scientists successfully transplanted genetically corrected muscle stem cells derived from people with type 2D limb-girdle
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July 01, 2012
Researchers are very good at understanding what is going on with things they can’t see directly, whether it be the membranes of muscle cells or the motor neurons of the spinal cord. But what is true
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July 01, 2012
An urgent need exists for biomarkers — biological indicators — in ALS research that can be used:
for diagnostic purposes;
to measure disease progression; and
to reliably assess the effects of
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July 01, 2012
Bryan Traynor
Even as some scientists work to develop animal and human cellular models of C9ORF72-related ALS (see C9ORF72 Research Models), others already are targeting the gene with experimental
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July 01, 2012
Phillip Wong, professor in the departments of pathology and neuroscience at Johns Hopkins University School of Medicine in Baltimore, is working — with MDA support — to develop research mouse models
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June 28, 2012
Eplerenone, a drug commonly used to prevent scarring after a heart attack, is being tested in a phase 2-3 clinical trial to determine whether it can stop or slow heart damage in people with Duchenne
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June 28, 2012
PTC begins non-US study of ataluren in DMD/BMD
Biopharmaceutical company PTC Therapeutics announced June 27, 2012, that it has initiated a non-U.S., open-label study of its experimental drug ataluren
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June 27, 2012
Treatment with an engineered version of the naturally produced interferon gamma protein enhanced the ability to move, and improved balance and coordination in mice with a disease resembling
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June 24, 2012
Update (Aug. 14, 2012): The Aug. 11 conference was attended by approximately 100 people with BMD and their families and friends, as well as some 40 clinicians and researchers. In addition, more than
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June 21, 2012
An experimental drug called RTC13, designed to treat Duchenne muscular dystrophy (DMD) by restoring production of the muscle protein dystrophin, has shown promise in experiments in dystrophin-
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June 21, 2012
Enhancing collaboration and transfer of technology between academia and industry was the subject of MDA's recent Translational Research Symposium, one of a series of four MDA-sponsored research
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June 18, 2012
The anti-diabetic drug pioglitazone did not improve survival time when tested as an add-on therapy to riluzole in a phase 2 clinical trial of people with amyotrophic lateral sclerosis (ALS).
The
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June 14, 2012
Update (July 10, 2012) — This story was updated to reflect the availability of a podcast with MDA research grantee Emanuela Gussoni, who discusses the development of stem-cell-based treatments for
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June 01, 2012
Update (Aug. 15, 2012): GlaxoSmithKline's exon-skipping drug GSK2402968 has been given a new generic name, "drisapersen."
Pharmaceutical company GlaxoSmithKline has announced the opening of a U.S.-
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May 31, 2012
The contribution of nervous system support cells called glia to the degeneration of motor neurons in amyotrophic lateral sclerosis (ALS)was the topic of serious discussion among researchers at MDA's
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May 30, 2012
Recently, MDA grantee Howard Worman at Columbia University, and colleagues, published encouraging results about the effects of a drug called selumetinib on the hearts of mice with a genetic mutation
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May 22, 2012
Overcoming barriers to gene therapy — the delivery of therapeutic genes — to treat neuromuscular diseases was the topic of a symposium jointly sponsored by MDA and the Association Française Contre
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May 18, 2012
Update (June 1, 2012): This story was updated to include the availability of a podcast on this topic.
An MDA-supported study has shown that an experimental drug called selumetinib, given to mice with
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May 17, 2012
This month, MDA is launching a new U.S.-based symposium series to address important topics and ultimately to accelerate therapy development for neuromuscular diseases.
"MDA’s new symposium series is
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