BioMarin's Morquio A drug confers "modest" benefit: FDA review

November 15, 2013 1:41 PM

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BioMarin's Morquio A drug confers "modest" benefit: FDA review

(Reuters) - An experimental drug to treat a rare genetic disorder that causes skeletal malformation and a host of related lung, eye ear and heart problems confers "modest" benefit, according to an initial review by the U.S. Food and Drug Administration.

The drug, Vimizim, is being developed by BioMarin Pharmaceutical Inc to treat Morquio A Syndrome, one of a group of lysosomal storage disorders known as mucopolysaccharidoses (MPS). Morquio A Syndrome is also known as MPS IV-A.

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