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FDA Approves First Drug to Treat Extremely Rare Enzyme Disorder

The near 150 people worldwide with MPS VII have had no option for treatment until now

The U.S. Food and Drug Administration has granted Ultragenyx Pharmaceutical, Inc. approval of MEPSEVII, a drug that treats an inherited metabolic condition called mucopolysaccharidosis type VII (MPS VII). Under 150 people worldwide are diagnosed with MPS VII, an extremely rare and progressive condition that affects most tissues and organs.   MPS VII is a lysosomal storage disorder caused by deficiency of an enzyme called beta-glucuronidase, which causes an abnormal buildup of toxic mater...

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