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Research News

[title]
Lithium Not Helpful in MDA-Supported Trial
May 01, 2010
An MDA-supported, 10-center trial of 108 people with ALS who took the oral medication lithium carbonate — some in conjunction with the ALS drug riluzole — has found the drug does not slow ALS
ALS Clinical Trials May-June 2010
May 01, 2010
Cytokinetics seeks participants for CK-2017357 study Cytokinetics, a South San Francisco biotechnology company, is seeking participants with ALS for a trial of its experimental compound CK-2017357.
ALS Research Roundup May-June 2010
May 01, 2010
IMMUNE SYSTEM MODULATOR HERALDS NEW STRATEGY IN ALS RESEARCH Blocking a key molecular pathway that the body uses to amplify an immune response has been found to delay disease onset and extend
[title]
ALS: Not Just About Motor Neurons Anymore
May 01, 2010
Motor neurons in the spinal cord, which are known as “lower” motor neurons, have long axons (fibers) through which they send signals to muscle fibers in the limbs, trunk, face and other parts of the
[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]
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]
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]
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]
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
[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]
MDA Awards Two New FSHD Grants
April 05, 2010
MDA has awarded two grants for research aimed at determining the precise molecular causes of facioscapulohumeral dystrophy (FSHD), and developing therapies for the disease. MDA and Friends of FSH
[title]
Familial ALS Research Update
April 02, 2010
The state of research in the familial (inherited) form of ALS was the focus of a special one-hour Webinar presented by the ALS Therapy Development Institute (ALS TDI) on March 25, with reports by
[title]
ALS Research: Disconnecting the Immune System
March 29, 2010
Blocking a key molecular pathway that the body uses to amplify an immune response has been found to delay disease onset and extend survival in mice with a disease that mimics human amyotrophic
[title]
Tadalafil Trial in Becker MD
March 24, 2010
Update (June 27, 2012): As of June 2012, this trial is ongoing but is no longer recruiting participants. The oral drug tadalafil (brand name Cialis), commonly used to treat erectile dysfunction, is
[title]
SMA Research: Saving Shortened SMN Protein
March 17, 2010
A research team at the University of Pennsylvania in Philadelphia has characterized the mechanism responsible for rapid decay of the survival of motor neurons (SMN) protein that is encoded by the
[title]
ALS SOD1 Trial: A ‘Watershed Moment’
March 05, 2010
Isis Pharmaceuticals of Carlsbad, Calif., has begun a phase 1 clinical trial of its experimental compound ISIS-SOD1-Rx in people with familial (inherited) ALS caused by toxic SOD1 protein molecules
[title]
Gene Therapy Rescues Mice with SMA
March 01, 2010
Scientists at four U.S. institutions have successfully used gene therapy to treat very young mice with a disease resembling severe spinal muscular atrophy (SMA).  Study results were published online
[title]
ALS TDI: Moving Forward on Multiple Fronts
March 01, 2010
In January 2007, MDA and the nonprofit biotech ALS Therapy Development Institute (ALS TDI) of Cambridge, Mass., launched a three-year, $36 million drug discovery partnership. In January 2010, MDA
ALS Research Roundup March-April 2010
March 01, 2010
This article reports news about clinical trials in ALS as of March 1, 2010, including: high-calorie diets, SOD1 blocker, altered communication, ceftraxione   Value of high-fat, high-calorie diets to
Letter to the Editor
March 01, 2010
In your January-February [2010] issue, I found your article about Gulf War ALS (“Research Roundup: Soil bacteria implicated in ALS-Gulf War connection”) very interesting. My husband Randy is one of
[title]
SMA Therapeutics: Changing the Code
February 26, 2010
 A process called trans-splicing has been shown to increase levels of a needed protein in mice with a disease resembling severe human spinal muscular atrophy (SMA), says a research team at the
[title]
Cardiomyopathy in Becker MD
February 12, 2010
This story was updated Oct. 6, 2010. In many forms of muscular dystrophy, including Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), weakness and degeneration of the cardiac
[title]
New Muscle Stem Cell Found in Mice
February 09, 2010
MDA-supported scientists in France have identified a previously unknown type of muscle stem cell located in the spaces between muscle fibers in mice. They say the new cells, dubbed "PICs," may play
[title]
ALS TDI: Full Speed Ahead
January 27, 2010
Significant progress on multiple fronts was reported by ALS TDI (the Amyotrophic Lateral Sclerosis Therapy Development Institute) in its first Webcast of 2010, hosted on Jan. 14. The Webcast
[title]
DMD, BMD: Trial of Ataluren in Nonwalkers
January 19, 2010
Update (March 3, 2010): An update to the article Ataluren Results Disappointing was posted. MDA has provided a grant of up to $1 million to the biotech firm PTC Therapeutics for a clinical trial of

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