In 2016, Spinraza became a game-changer for spinal muscular atrophy (SMA) patients. It was the first FDA-approved treatment for the neurodegenerative disease, which is the leading genetic cause of infant death. The drug ...
Jul 13, 2022
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Up to 45% of patients with the most common inherited neuromuscular disease could benefit from a new "cocktail" drug being developed at the University of Alberta, according to research published this week in the Proceedings ...
Feb 23, 2022
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Using a short, synthetic chain of chemically modified nucleotides engineered in the RNA Therapeutics Institute at UMass Chan Medical School, Robert H. Brown Jr., DPhil, MD, Jonathan Watts, Ph.D., and colleagues have shown ...
Dec 23, 2021
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Alexander disease is a progressive and rare neurological disorder with no cure or standard course of treatment. But a new study led by researchers at the University of Wisconsin–Madison involving a rat model of the disease ...
Nov 17, 2021
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Many patients with multiple myeloma, a type of blood cancer, eventually develop resistance to one treatment after another. That's in part because cancer stem cells drive the disease—cells that continually self-renew. If ...
Jan 20, 2021
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A study from a team of Massachusetts General Hospital (MGH) investigators points toward a potential strategy for treating X-linked disorders - those caused by mutations in the X chromosome - in females. Their report published ...
Jan 4, 2018
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In two studies of mice, researchers showed that a drug, engineered to combat the gene that causes spinocerebellar ataxia type 2 (SCA2), might also be used to treat amyotrophic lateral sclerosis (ALS). Both studies were published ...
Apr 12, 2017
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In a study of mice and monkeys, National Institutes of Health funded researchers showed that they could prevent and reverse some of the brain injury caused by the toxic form of a protein called tau. The results, published ...
Feb 8, 2017
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Under ordinary circumstances, the protein tau contributes to the normal, healthy functioning of brain neurons. In some people, though, it collects into toxic tangles that damage brain cells. Such tangles are a hallmark of ...
Jan 25, 2017
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Ottawa scientists have discovered a trigger that turns muscle stem cells into brown fat, a form of good fat that could play a critical role in the fight against obesity. The findings from Dr. Michael Rudnicki's lab, based ...
Feb 5, 2013
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