Fabry disease: FDA drafts drug development guidance
The FDA on Wednesday issued draft guidance providing recommendations to drugmakers on clinical trial design and eligibility criteria for products to treat Fabry disease.
Fabry disease is a rare inherited disorder caused by a deficiency of the lysosomal enzyme alpha-galactosidase A (α-Gal A). According to the FDA, patients with Fabry disease experience chronic gastrointestinal and neuropathic symptoms and progressive organ damage that eventually causes chronic renal disease, renal failure, cardiovascular disease and strokes.
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