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Research News

[title]
Second Phase 2 Trial of CK-2017357 Under Way
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 (
Figuring Out FUS
July 01, 2011
How to prevent selective loss of neurons? Benjamin BrooksCarolinas Medical CenterCharlotte, N.C Brooks, director of the MDA/ALS Center at Carolinas Medical Center, and colleagues are interested in
[title]
ALS Research Roundup July-August 2011
July 01, 2011
Eating and breathing: A possible 'synergistic' effect in ALS Initial results of a multicenter pilot study to evaluate nutritional requirements and early intervention for breathing problems in ALS are
[title]
FUS on the Loose
July 01, 2011
In 2009, it was discovered that genetic mutations in the fused in sarcoma gene— FUS for short — were linked to some cases of ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease). Left — FUS
[title]
ALS Research Briefs: Ataxin 2 Gene
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 (
[title]
Stem Cell Model Creates 'ALS in a Dish'
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 “
[title]
Researchers Studying DMD-Affected Families
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 (
[title]
Research Briefs: DMD, BMD Trials Update
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
[title]
Safety Stem Cell Trial in ALS Set to Advance
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 (
[title]
Research Briefs: Stem Cells
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
[title]
Research Briefs: The DMD/BMD-Affected Heart
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
[title]
Researchers Exploring Disability Perceptions
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
[title]
MDA Awards Grant to Test MTM Gene Transfer in Dogs
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
[title]
FDA Approves Phase 1 Clinical Trial of RG3039 in SMA
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
[title]
ALS Research Briefs
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
[title]
Research Briefs: BMD, DMD, EDMD, FA, LGMD, OPMD, Pompe disease, SMA
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
[title]
FUS and ALS: What's the Connection?
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
[title]
ACE-031 Clinical Trials in Duchenne MD Stopped for Now
May 03, 2011
Biopharmaceutical companies Acceleron Pharma and Shire announced April 21, 2011, that MDA-supported clinical trials of ACE-031 for Duchenne muscular dystrophy (DMD) have been halted. A phase 2, dose-
[title]
Covering a Broad Canvas: The Search for ALS Treatments
May 01, 2011
ALS is a disease that holds tight to its many secrets, even after decades of relentless pressure by dedicated and well-qualified researchers. Part of the complexity of ALS is that it involves
[title]
ALS Research Roundup May-June 2011
May 01, 2011
Reactivated virus may contribute to ALS Autopsy samples have shown a viral protein called HERV-K reverse transcriptase is much more prevalent in ALS brain samples than in brain samples obtained from
[title]
ALS Research Briefs
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
[title]
ISIS-SOD1-Rx: So Far, So Good
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
[title]
ALS TDI Webinar Discusses Current Research
April 21, 2011
The state of the science in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease), current lines of research, efforts to establish a TDP43 research mouse colony, and a primer on protein-based
[title]
AAN Research Briefs on DM, DMD, LGMD, MG, MMD, SMA
April 20, 2011
Below are brief reports and links to more information about neuromuscular disease research presented at the 63rd annual meeting of the American Academy of Neurology (AAN), held in Honolulu April 9-16
[title]
ALS Research Briefs from 2011 AAN Meeting
April 15, 2011
Neuralstem's trial of spinal cord injections of stem cells is on track The first nine participants in a phase 1 (safety and feasibility) trial of neural stem cell transplantation in amyotrophic

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