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Home › Posts tagged Mucopolysaccharidosis Type IVA (Morquio A syndrome)

Tag: Mucopolysaccharidosis Type IVA (Morquio A syndrome)

FDA approves Elosulfase Alfa [Vimizim] to treat the rare congenital enzyme disorder Mucopolysaccharidosis Type IVA (Morquio A syndrome)

By Joseph Gut - thasso Posted on April 18, 2014 Posted in Congenital, Genetic Background, New Drug Approval, Pediatrics, Personalized Medicine, Thasso Post, Theragenomics No Comments Tagged with American Food & Drug Administration (FDA), Elosulfase Alfa [Vimizim], Mucopolysaccharidosis Type IVA (Morquio A syndrome), N-Acetylgalactosamine-6-sulfate Sulfatase (GALNS), Pediatric Patients, Rare Pediatric Disease Priority Review Voucher Program

April 18, 2014 – Have you ever heard of Mucopolysaccharidosis Type IVA (Morquio A syndrome)? Most probably not. And I would also think that only very few people are familiar with the “Rare Pediatric Disease Priority Review Voucher Program” by …

FDA approves Elosulfase Alfa [Vimizim] to treat the rare congenital enzyme disorder Mucopolysaccharidosis Type IVA (Morquio A syndrome) Read more »

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