For which of the following diseases is enzyme replacement therapy available?
Correct Answer: Gaucher's disease
Description: Gaucher's disease is the first storage disorder for which the treatment became available. Cerezyme' a recombinantly produced acid b-glucosidase is being currently used. Ref: Harrison's Principles of Internal Medicine, 17th Edition, Page 2455 ; O P Ghai Essential Pediatrics, 6th Edition, Pages 616-617
Category:
Pediatrics
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