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Report on the International ALS/MND Symposium Hosted by The ALS Association
Over 800 researchers and clinicians converged on Orlando for the 21st Annual ALS/MND Symposium, sponsored by the Motor Neurone Disease Association, and hosted by The ALS Association. The meeting is one of the largest meetings in the world devoted to ALS research and treatment.
The ALS Association was represented by President and CEO Jane Gilbert, Chief Scientist Lucie Bruijn, Ph.D., Vice President of Patient Services Sharon Matland, and three ALS Association National Trustees: Ellyn Phillips, Elizabeth Rosenberg and Cynthia Douthat. During the meeting of the International Alliance of ALS/MND, which was held just prior to the symposium also in Orlando, Sharon Matland was elected to the board of the International Alliance.
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Cytokinetics Reported on “Evidence of Effect” Clinical Trial at ALS Association Hosted ALS/MND International Symposium
On Monday, Dec. 13, 2010, Cytokinetics, Inc. announced the successful completion of its Phase IIA “Evidence of Effect” clinical trial of CK-2017357 in patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, during an oral presentation included in the Clinical Trials Session at the 21st International ALS/MND Symposium in Orlando, Fla.
A presentation titled, “A Phase 2A, Double-Blind, Randomized, Placebo-Controlled, Single-dose, Crossover Study of the Selective Fast Skeletal Muscle Troponin Activator, CK-2017357, in Patients with ALS” was made by Jeremy M. Shefner, M.D., Ph.D., Professor and Chair of the Department of Neurology at the Upstate Medical University at the State University of New York. CK-2017357, a fast skeletal muscle troponin activator, is the lead drug candidate from the company's skeletal muscle contractility program. CK-2017357 selectively activates the fast skeletal muscle troponin complex by increasing its sensitivity to calcium, leading to an increase in skeletal muscle force.
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ALS Association-Funded Research Reveals VCP Mutations as a Cause of Familial ALS
Using exome sequencing, which is functionally the most relevant part of the genome, scientists identified an amino acid change in the valosin-containing protein (VCP) gene in an Italian family published in the December 9, 2010, edition of Neuron Report. In this study of this family, SOD1, TDP-43, and FUS mutations were previously excluded in an attempt to identify the underlying genetic abnormality responsible for the disease.
“It is very exciting to see that this new sequencing technology has resulted in a significant finding for ALS,” said ALS Association Chief Scientist Lucie Bruijn, Ph.D. “Discovery of VCP provides another piece of the puzzle in understanding the disease mechanism.”
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Persons with Amyotrophic Lateral Sclerosis (ALS) Wanted for a Research Study
Researchers at Penn State College of Medicine are seeking participants for a research study on Quality of Life (QOL) and ALS. The purpose of the study is to better understand the QOL of patients with ALS who receive different forms of care for the disease.
Participants will complete a questionnaire (online or with a paper and pencil version) that asks questions about physical health status, quality of life, and use of medical services for care of ALS. Survey responses are confidential. All persons who have been diagnosed with the disease Amyotrophic Lateral Sclerosis are eligible to participate in this study.
To access the online questionnaire, go to our ALS Clinic website: http://www.alsphiladelphia.org/pennstatehershey
For more information call the study coordinator, Beth Stephens, at 717-531-0003, extension 283395, or by email at [email protected] Study Director: Zachary Simmons, MD, Department of Neurology, Penn State College of Medicine.
‘This research study has been approved by the Institutional Review Board, under federal regulations, at Penn State College of Medicine, Penn State Hershey Medical Center.’ | |
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