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Press Releases
Enzyme Therapy Shown Effective and Safe for Fabry DiseaseTuesday, June 05, 2001Enzyme replacement therapy effectively and safely reduces neuropathic pain in patients with Fabry disease, results of a double-blind placebo-controlled clinical trial show. The therapy also corrects the underlying metabolic defect in patients' cells and improves their heart and kidney functions. This is the first published report to show significant clinical benefits from a controlled study of enzyme replacement therapy for Fabry disease.
Fact Sheet
Gene for Last Major Form of Batten Disease DiscoveredFriday, September 19, 1997Just two years ago, the origins of the fatal childhood neurological disorders called Batten disease were shrouded in mystery, and there were few prospects for effective treatment. Now, for the first time, researchers can describe the genetic underpinnings of all major childhood forms of the disease.
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