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PTC Therapeutics Enters Orphan Disease Pact

Phase II study will evaluate the safety and pharmacokinetics of Translarna in MPS I

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By: Kristin Brooks

Managing Editor, Contract Pharma

PTC Therapeutics, Inc. entered a collaboration with the Orphan Disease Center at the Perelman School of Medicine at the University of Pennsylvania focused on translational research for discovering and developing new treatments for orphan disorders.   The first initiative will focus on Translarna (ataluren) for the lysosomal storage disease Mucopolysaccharidosis I (MPS I) due to a nonsense mutation. MPS I is an inherited disorder caused by a deficiency of alpha-L-iduronidase, where cells are unab...

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