News tagged with enzyme replacement therapy
Anti-cancer drug fights immune reaction in some infants with Pompe disease
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 ...
Genetics
Oct 11, 2012 |
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Enzyme replacement therapy shows promising results in X-linked myotubular myopathy
A collaborative research team including a Medical College of Wisconsin (MCW) pediatric neuropathologist successfully mitigated some of the effects of a muscular disease by using a new targeted enzyme replacement therapy strategy ...
Genetics
Jan 22, 2013 |
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The same genetic defect causes Pompe disease in both humans and dogs
Pompe disease, a severe glycogen storage disease appearing in Lapphunds is caused by a genetic defect in acid α-glucosidase gene. The same genetic mutation also causes the equivalent disease in humans. Based ...
Genetics
Feb 15, 2013 |
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