News tagged with ptc124
New findings raise questions about process used to identify experimental drug
A study by National Institutes of Health (NIH) researchers has revealed surprising new insights into the process used to initially identify an experimental drug now being tested in people with cystic fibrosis and muscular ...
Feb 03, 2009 |
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Treatment approach to human Usher syndrome: Small molecules ignore stop signals
Usher syndrome is the most common form of combined congenital deaf-blindness in humans and affects 1 in 6,000 of the population. It is a recessive inherited disease that is both clinically and genetically ...
Jul 01, 2011 |
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Latest advances in gene therapy for ocular disease are highlighted in Human Gene Therapy
Disorders of the eye are excellent targets for gene therapy because the ocular environment is readily accessible, relatively easy to monitor, and sequestered from the rest of the body. A series of articles ...
May 09, 2011 |
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New hope exists in treating inherited disease by suppressing DNA mutations
Genetic mutation can disrupt the way human cells make proteins, which in turn leads to inherited disease. David Bedwell, a professor in the University of Alabama at Birmingham (UAB) Department of Microbiology, says scientists ...
Apr 26, 2010 |
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Drug fights cystic fibrosis
An experimental drug that has proven effective in treating muscular dystrophy also works for cystic fibrosis, according to researchers at the University of Alabama at Birmingham (UAB).
Medicine & Health / Medications
Feb 05, 2008 |
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