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