First drug for Morquio A syndrome gains FDA OK

02/16/2014 | Reuters · Medscape (free registration)

BioMarin Pharmaceutical obtained FDA approval to market Vimizim, or elosulfase alfa, as a treatment for Morquio A syndrome. Vimizim is the first drug approved for the rare genetic condition. Due to risk of anaphylaxis, the drug will carry a boxed warning. About 800 people in the U.S. are affected by the condition, characterized by deficiency of an enzyme that leads to excessive storage of a certain sugar.

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Reuters · Medscape (free registration)

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