Research News
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August 08, 2011
The administration of a protein called prolactin has been shown to slow weight loss, improve motor function and increase life span by approximately 70 percent in mice with a disease resembling a
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August 05, 2011
The 2011 Periodic Paralysis Association Conference will be held Nov. 4-6 in Orlando, Fla., and is geared toward people with periodic paralysis (PP), their families, physicians and friends.
This is
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August 04, 2011
The "window of opportunity" for treating infants at risk of developing spinal muscular atrophy (SMA) may not be as narrow as some experts have feared, new experiments in mice suggest.
MDA research
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July 28, 2011
An experimental compound called trichostatin A (TSA) has been shown to improve nerve-cell survival, increase strength and muscle weight, and result in longer life span in mice with a disease
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July 26, 2011
Complete and extremely encouraging findings from a phase 1b-2 trial of eteplirsen (AVI-4658), an exon skipping drug in development to treat a portion of the Duchenne muscular dystrophy (DMD)
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July 22, 2011
Biotechnology company BrainStorm Cell Therapeutics has announced plans to collaborate with two American institutions to test its experimental stem cell technology in people with amyotrophic lateral
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July 20, 2011
A one-year, MDA-supported study comparing a weekend-only prednisone treatment schedule with a daily prednisone schedule in boys with Duchenne muscular dystrophy (DMD) has found that the two treatment
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July 14, 2011
In hopes of collecting as much information as possible, the National ALS Registry, which opened in October 2010, already is expanding its reach.
Registry officials have made the enrollment process
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July 07, 2011
Edison drugs target FA, mitochondrial diseases
Edison Pharmaceuticals, a biotechnology company, announced in June 2011 that its experimental drug EPI-A0001 has shown promise in a 28-day, placebo-
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July 01, 2011
San Francisco biotechnology company Cytokinetics announced June 21, 2011, that it has opened enrollment for a new clinical trial of CK-2017357 at three different dosage levels in people with ALS (
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June 28, 2011
In a 2010 study, scientists working in the United States and Germany found that small polyglutamine ("polyQ")expansions of DNA in the ataxin 2gene significantly increase the risk for developing ALS (
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June 23, 2011
A human cellular model of ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease) has been created using cells taken from people with a subtype of the disease called ALS8.
This so-called “
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June 22, 2011
Belen Pappa, a graduate student in genetic counseling, is seeking participants for a survey-based study about psychosocial functioning in families in which a child has Duchenne muscular dystrophy (
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June 21, 2011
U.S. trial of eteplirsen for Duchenne MD delayed
AVI BioPharma of Bothell, Wash., announced June 9, 2011, that the launch of its phase 2 trial of eteplirsen (pronounced eh-TEHP-lur-son; formerly
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June 17, 2011
Maryland biotherapeutics company Neuralstem has announced favorable results from the first part of a phase 1 trial to test the safety of its experimental spinal cord stem cell treatment in ALS (
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June 13, 2011
Stem cells are a hot topic these days in medicine, science and law, although the term has multiple meanings and it's easy to get confused.
In short, stem cells are cells at an early stage of
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June 06, 2011
In both Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), deterioration of the heart muscle, a condition known as cardiomyopathy, is a major cause of disability and death.
DMD
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June 01, 2011
Researchers at the Psychology of Disability Lab at the University of Michigan in Ann Arbor are exploring the social identity of people with disabilities through a short, anonymous, Web-based
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May 26, 2011
MDA’s translational research program has announced it is funding research into a potential treatment for the inherited muscle disorder X-linked myotubular myopathy (MTM).
The grant of $369,365 grant
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May 20, 2011
In a historic first, biotech company Repligen Corp., of Waltham, Mass., has received approval from the U.S. Food and Drug Administration (FDA) to begin a phase 1 clinical trial of the experimental
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May 19, 2011
NurOwn stem cell therapy trial set to begin
Biotechnology company Brainstorm Cell Therapeutics announced May 17, 2011, that it has received approval from Israel's Ministry of Health to conduct a
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May 16, 2011
Idebenone may help maintain respiratory function in DMD
Santhera Pharmaceuticals announced May 9, 2011, that its drug Catena (generic name idebenone) appears to slow the decline in respiratory
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May 12, 2011
The gene for FUS was associated in 2009 with some forms of ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease).
Now, overlapping findings from four recent studies have revealed tantalizing
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April 29, 2011
Patients Like Me online lithium study utilized social media
An observational study of data self-reported on the online forum Patients Like Me by people with ALS who elected to take the drug lithium
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April 27, 2011
Early results of a phase 1 clinical trial of ISIS-SOD1-Rx, an antisense oligonucleotide drug for SOD1-related amyotrophic lateral sclerosis (ALS), show the drug appears to be safe and well tolerated
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