Research News
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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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April 02, 2013
An online survey about ALS symptoms, now open to people with amyotrophic lateral sclerosis (ALS), is being conducted by the Neurological Clinical Research Institute at Massachusetts General Hospital
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April 01, 2013
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Winter 2013 MDA research grants
$13.6 Million in New MDA Grants Promote Understanding, Treatment of Neuromuscular
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April 01, 2013
The approval of new drugs in the United States is a complex process that seeks to balance the need to protect the health and safety of the population with the need to provide treatment as soon as
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April 01, 2013
Merit Cudkowicz
Put together the words amyotrophic lateral sclerosis (ALS) and the phrase clinical trials expert, and one name that is sure to follow is Merit Cudkowicz.
Cudkowicz is chief of
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March 29, 2013
Scientists continue to work at uncovering the biological mechanisms underlying amyotrophic lateral sclerosis (ALS). One area of intense study involves the potential role of central nervous system
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March 26, 2013
Drug development and identifying new leads for possible drug development are in the news for five neuromuscular diseases in MDA’s program.
DMD: Building muscle, fighting inflammation
DART
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March 22, 2013
The latest advances in brain and central nervous system research were discussed at the 65th annual meeting of the American Academy of Neurology, held in San Diego, March 16-23, 2013.
Reports included
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March 21, 2013
An experimental drug designed to treat the underlying molecular defect in spinal muscular atrophy (SMA) has shown encouraging results in a phase 1 trial.
The drug, ISIS-SMNRx, is being developed and
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March 20, 2013
A therapeutic strategy that combines gene therapy and stem cell transplantation has shown encouraging results in mice with a disorder mimicking Duchenne muscular dystrophy (DMD).
"Our findings
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March 20, 2013
Proposals exploring the feasibility and advisability of implementating newborn screening for two disorders in MDA's program — Duchenne muscular dystrophy and Pompe disease— were presented to a
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March 15, 2013
Advances in technology have led to greater sensitivity in imaging techniques, increasing scientists' ability to see into the brain and spinal cord. Now, a research team reports that a technique
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March 14, 2013
Spinal muscular atrophy (SMA) is a disease in which nerve cells that control muscles (motor neurons) in the spinal cord die, causing progressive weakness in the voluntary muscles. Recent research
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March 12, 2013
An international patientregistry (database) is gathering information about children and adults with any form of congenital muscular dystrophy (CMD), a congenital myasthenic syndrome or a congenital
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March 08, 2013
The number of new cases of amyotrophic lateral sclerosis (ALS) identified each year (incidence) and the number of people living with the disease (prevalence) appears to be lower in American Indians
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March 03, 2013
Mice with a disorder mimicking human spinal-bulbar muscular atrophy(SBMA, or Kennedy disease) that were treated with an experimental therapy called arimoclomol showed improved nerve-cell survival,
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February 28, 2013
Idebenone (under the brand name Catena) in July 2008 received conditional market approval in Canada for the treatment of Friedreich's ataxia (FA). Now, based on additional data that fails to confirm
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February 27, 2013
Scientists at the biopharmaceutical company Genzyme, working with mice, say they have modified and improved an existing experimental strategy to treat type 1 myotonic muscular dystrophy (MMD1, also
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February 21, 2013
Trials of the experimental muscular dystrophy drug ataluren have shown that the drug is generally well-tolerated and has a positive effect on walking ability.
However, so far the efforts of
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February 15, 2013
The experimental drug AT2220 has shown benefit as an enhancer of enzyme replacement therapy for the metabolic muscle disorder Pompe disease (acid maltase deficiency).
The drug, a pharmacological
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February 15, 2013
Prednisone and other drugs in the corticosteroid family have become standard treatment for Duchenne muscular dystrophy (DMD) in much of the world for almost a decade. In this disorder, they slow the
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February 13, 2013
Researchers conducting a study of natural history and biomarkers in infants with type 1 spinal muscular atrophy (SMA) currently are recruiting participants at 15 locations across the United States
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February 11, 2013
MDA has awarded a $400,000 grant to National Institutes of Health (NIH) Laboratory of Neurogenetics researchers to perform exome sequencing on samples taken from 1,000 people with sporadic
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February 10, 2013
The U.S. Food and Drug Administration (FDA) has given the go-ahead to the nonprofit biotech ALS Therapy Development Institute (ALS TDI) to conduct a clinical trial of TDI132 — also known as
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