The influenza virus remains a worldwide threat to humans, causing an average of 36,000 deaths and 200,000 hospitalizations each year in the United States alone. As health care professionals prepare for another flu season, ...
Nov 17, 2011
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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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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 from the School of Medicine at The University of Texas Health Science Center at San Antonio have found evidence that directly links disrupted metabolism (energy production in cells) to a common and often fatal ...
Jul 17, 2015
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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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A first-in-man clinical trial, testing a newly developed drug for a rare genetic condition called mucopolysaccharidosis type II (MPS II) or Hunter syndrome, is underway at Emory University.
Jun 3, 2015
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Gene therapy might soon offer a new option for children with a rare genetic disorder that damages tissues throughout the body, researchers are reporting.
Nov 18, 2021
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Adding a third anti-cancer agent to a current drug cocktail appears to have contributed to dramatic improvement in three infants with the most severe form of Pompe disease—a rare, often-fatal genetic disorder characterized ...
Oct 11, 2012
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Kids with Pompe disease fail because of a missing enzyme, GAA, that leads to dangerous sugar build-up, which affects muscles and movement. An enzyme replacement treatment pioneered at Duke University has saved many lives, ...
Jan 5, 2012
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Researchers at UT Southwestern Medical Center have found that alternative splicing – a process that allows a single gene to code for multiple proteins – appears to be a new potential target for anti-telomerase cancer ...
Apr 19, 2013
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