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Fabry Disease Therapy

Researchers at Institute of Metabolic Disease release new data on fabry disease therapy

Published in Pharma Law Weekly, June 2nd, 2009

Research findings, 'Fabry disease,' are discussed in a new report. "Fabry disease, an X-linked disorder of glycosphingolipids that is caused by the deficiency of alpha-galactosidase A, is associated with dysfunction of many cell types and includes a systemic vasculopathy. As a result, patients have a markedly increased risk of developing small-fiber peripheral neuropathy, stroke, myriad cardiac manifestations and chronic renal disease," scientists writing in the journal Pharmacology and Therapeutics report.

"Virtually all complications of Fabry disease are non-specific in nature and clinically indistinguishable from similar abnormalities that occur in the context...

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