Enzyme 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.
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Just 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.

Treatment with enzyme replacement therapy reverses symptoms in patients with Type I Gaucher's disease, according to a study published in the May 23 issue of the New England Journal of Medicine* and conducted by scientists at the National Institute of Neurological Disorders and Stroke (NINDS). A rare metabolic disorder, Type I Gaucher's disease affects an estimated 10,000 to 12,000 Americans.
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