Last year a clinical trial of L-DOPA—a mainstay of Parkinson's disease therapy—was launched for Angelman syndrome, a rare intellectual disorder that shares similar motor symptoms such as tremors and difficulty with balance. ...
Nov 12, 2012
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Children born with rare, inherited conditions known as Congenital Disorders of Glycosylation, or CDG, have mutations in one of the many enzymes the body uses to decorate its proteins and cells with sugars. Properly diagnosing ...
May 10, 2013
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A novel type of therapy, known as ANGPTL3 inhibitor therapy, was effective in lowering triglycerides in certain types of patients with severe hypertriglyceridemia (sHTG) who had a prior episode(s) of acute pancreatitis. sHTG ...
Mar 9, 2023
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Whitehead Institute researchers have determined the mechanism used by c-Myc to increase the expression of all active genes in cancer cells. Elevated levels of c-Myc are linked to increased rates of metastasis, disease recurrence, ...
Sep 27, 2012
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Atherosclerosis is an inflammatory disease with accumulation of cholesterol in the vessel walls. The atherosclerotic plaque is built up throughout life and when it ruptures it leads to heart attack or stroke. T cells are ...
Jul 31, 2013
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University of Michigan researchers have determined how a gene that is known to be defective in Down syndrome is regulated and how its dysregulation may lead to neurological defects, providing insights into potential therapeutic ...
Jun 5, 2013
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The Malt1 protein is one of the most important control centers in human immune cells and a real all-rounder. Genetic defects in it can lead to the development of lymphatic cancer (lymphoma). A possible therapeutic approach ...
Nov 17, 2014
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Massachusetts General Hospital (MGH) researchers have identified a gene variant that helps predict how much weight an individual will lose after gastric bypass surgery, a finding with the potential both to guide treatment ...
May 2, 2013
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Having identified the important role in controlling insulin secretion played by the protein Cx36, a team of scientists at the University of Geneva have perfected an innovative method which enables testing the effectiveness ...
Jul 26, 2012
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Caring for a patient with sepsis requires walking a treatment tightrope. Clinicians must identify the pathogen that is causing a patient's infection, carefully monitor the patient's response to antibiotics and supportive ...
Nov 11, 2019
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