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Research News

[title]
It's Myasthenia Gravis Awareness Month
June 16, 2010
In recognition of June being National Myasthenia Gravis Awareness Month, Quest News Online is examining the disease from several perspectives. This article offers an overview of signs, symptoms,
[title]
The Flip of a Switch
June 15, 2010
Neuraltus Pharmaceuticals of Palo Alto, Calif., is developing a small molecule whose target is regulation of immune system cells believed to contribute to neuroinflammation and disease progression in
[title]
ALS Ceftriaxone Trial Still Open
June 11, 2010
A 600-person trial of intravenous ceftriaxone, an antibiotic in the cephalosporin family that's approved to treat certain types of infections, is still looking for participants at 53 sites in the
[title]
OPMD: Cystamine Strengthens Muscles in Mice
June 09, 2010
Scientists in the United Kingdom have found that mice carrying a genetic mutation that causes oculpharyngeal muscular dystrophy (OPMD) in humans and showing a disease resembling human OPMD benefited
[title]
DMD Trial: AVI4658 Increased Dystrophin Production
June 02, 2010
The biopharmaceutical company AVI BioPharma has announced additional encouraging results from its clinical trial of AVI4658, an experimental treatment for Duchenne muscular dystrophy (DMD). The new
[title]
ALS TDI: Changes in the Works
June 01, 2010
In its quarterly Research Update Webcast on May 18, 2010, the ALS Therapy Development Institute (ALS TDI) informed viewers of a "paradigm shift" in TDI research. The changes include the addition of
[title]
FA Research: Idebenone Strikes Out Again
May 28, 2010
Idebenone, a substance believed to act as an antioxidant and to aid in energy production in cellular structures called mitochondria, has failed to benefit people with Friedreich's ataxia (FA) in a
[title]
Podcasts Focus on Latest Neuromuscular Research
May 24, 2010
Scientists at the Neuromuscular Disorders Program at Nationwide Children’s Hospital in Columbus, Ohio, are producing a series of podcasts examining current research in neuromuscular disease. The
[title]
Gene Therapy Success in IBM
May 21, 2010
Scientists at five U.S. institutions have successfully used gene therapy to improve muscle function in a single human subject with a hereditary form of inclusion-body myositis (IBM) caused by
[title]
SOD1 Versus Other ALS: Apples and Oranges?
May 19, 2010
Scientists at Northwestern University Feinberg School of Medicine in Chicago have announced new findings showing some forms of familial and nonfamilial ("sporadic") amyotrophic lateral sclerosis (ALS
[title]
DMD Research: New Funding for Imaging Studies
May 17, 2010
The use of magnetic resonance imaging (MRI) as an assessment tool in boys with Duchenne muscular dystrophy (DMD) is being studied by former MDA grantee Krista Vandenborne, through a $7.5 million
[title]
MTM: Unlocking Muscle Fibers to Myotubularin
May 05, 2010
MDA-supported researchers at 4s3 Bioscience, a biotechnology company in Medford, Mass., are using a new molecular strategy to transport a potentially therapeutic protein into muscles, as an
[title]
New Tools for Trials in Children with CMT
April 23, 2010
Two scales that can be used to assess disease progression and any response that may occur to a treatment have been developed for children with Charcot-Marie-Tooth (CMT) disease. One, called the CMT
[title]
Curcumin Helps Mice With CMT-Like Disease
April 21, 2010
Mice with a genetic mutation in the myelin protein zero (MPZ) gene, which develop a disease resembling human type 1B Charcot-Marie-Tooth disease (CMT1B), benefited from treatment with curcumin and
[title]
NT3 Gene Therapy for CMT1A Benefits Mice
April 21, 2010
Mice with a disorder resembling type 1A Charcot-Marie-Tooth (CMT1A) disease that received a single intramuscular injection of genes for the protein neurotrophin 3 (NT3) showed improvements in grip
[title]
Caution: Immune Response Seen in DMD Gene Therapy
April 21, 2010
Unwanted responses by the immune system to dystrophin have been seen in a small, MDA-supported clinical trial of gene therapy for Duchenne muscular dystrophy (DMD)— an unexpected finding,
[title]
DMD/BMD: Taking a Closer Look at Ataluren
April 19, 2010
PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, has announced findings that reflect the company's closer look at a large-scale trial of its experimental drug ataluren. The
[title]
Luring Away Myostatin Can Boost Muscle Size
April 16, 2010
ACE031, a laboratory-modified protein developed by Acceleron Pharma of Cambridge, Mass., has shown promise as a therapy to increase muscle mass, based on results of a trial in healthy volunteers. The
[title]
SMN Gene Transfer Benefits Mice
April 15, 2010
A research group from the University of Sheffield in the United Kingdom has found that mice with a disease mimicking human spinal muscular atrophy (SMA) benefited significantly from intravenous
[title]
More Good News about Exon Skipping
April 15, 2010
AVI4658, an experimental treatment for patients with Duchenne muscular dystrophy (DMD) caused by certain mutations in the gene for the muscle protein dystrophin, has shown promising results when
[title]
Narrowing Down CMT Subtypes
April 15, 2010
  Editor's note:This story was updated Feb. 4, 2011, to reflect the availability of a paper and editorial on this subject in Annals of Neurology. Results of genetic testing done at Wayne State
[title]
Progress in Exon Skipping for DMD
April 13, 2010
The multinational pharmaceutical company GlaxoSmithKline (GSK) and the Dutch biotechnology company Prosensa  announced "intriguing results" following a 12-person trial of an experimental "exon
[title]
Gentamicin Shows Mixed Results in DMD
April 13, 2010
Results of the MDA-supported trial of gentamicin in Duchenne muscular dystrophy (DMD) were presented Wednesday, April 14, at the annual meeting of the American Academy of Neurology (AAN), held in
[title]
MDA Study Shows Lithium Not Helpful in ALS
April 12, 2010
An MDA-supported, 10-center trial of more than 100 people with amyotrophic lateral sclerosis (ALS) who took the oral medication lithium carbonate — some in conjunction with the ALS drug riluzole —
[title]
ALS Research: Zenvia Improves Emotional Symptoms in ALS
April 12, 2010
The experimental drug Zenvia, developed by Avanir Pharmaceuticals as a treatment for unwanted laughing/crying spells in ALS, has shown continued promise in lessening the frequency of such episodes

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