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Translarna Granted Orphan Drug Designation In The U.S. And Europe For The Treatment Of Mucopolysaccharidosis I

2014-12-29 01:43:03| drugdiscoveryonline Home Page

PTC Therapeutics, Inc. (Nasdaq: PTCT) today announced that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted orphan-drug designation (ODD) to Translarna™ (ataluren) for the treatment of patients with Mucopolysaccharidosis I (MPS I)

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Category:Biotechnology and Pharmaceuticals

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