A targeted therapy, currently being studied for treatment of certain cancers including glioblastoma, may also be beneficial in treating other neurologic diseases, a study at the University of Cincinnati shows.
Apr 6, 2020
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A team at the University of Manchester have developed a novel stem cell gene therapy approach to treat children with a devastating genetic disease. The approach is currently being developed for clinical trial in patients ...
Jul 6, 2018
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Thanks to the sequencing of the human genome, scientists can now discover potential disorders for which there are no known patients. One such disorder is MPS III-E, originally also called Dierks's disorder after its discoverer. ...
Jan 8, 2018
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A new therapy involving stem cells may offer a better quality of life for patients with Hurler syndrome, a rare genetic disease that can cause crippling physical conditions and lead to early death.
Nov 16, 2017
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Researchers are working hard to fight childhood dementia and hope a new enzyme replacement therapy can help those unlucky enough to have such a debilitating disease.
Sep 1, 2017
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Two weeks and several political disasters ago, the House of Representatives passed the American Health Care Act of 2017, and soon lists of "pre-existing conditions" festooned news feeds. We all ticked off a few. But the lists, ...
May 22, 2017
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A team of Canadian physicians and researchers is believed to be the first in the world to have used gene therapy to treat a patient with Fabry disease, a rare inherited enzyme deficiency that can damage major organs and shorten ...
Feb 16, 2017
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After decades investigating a rare, life-threatening condition that cripples the muscles, Duke Health researchers have developed a gene therapy they hope could enhance or even replace the only FDA-approved treatment currently ...
Jan 26, 2017
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Researchers are closer to finding a better way to treat children with a rare metabolic disorder called MPS I. It is caused by a deficiency of the key enzyme IDUA needed to break down complex sugars in cells. MPS I eventually ...
Jul 28, 2016
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Researchers at Duke Health have identified a potential new avenue for treating Pompe disease, a rare condition caused by the build-up of glycogen, a storage form of sugar, in cardiac and skeletal muscle, the liver and other ...
Jul 27, 2016
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