Amicus Therapeutics, Inc. (FOLD) Announces Chaperone-Advanced Replacement Therapy in Development for Mucopolysaccharidosis Type I (MPS I)
6/25/2013 10:05:32 AM
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CRANBURY, N.J., June 25, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD) today disclosed a preclinical Chaperone-Advanced Replacement Therapy (CHART™) program for Mucopolysaccharidosis Type I (MPS I), a lysosomal storage disease caused by missing or deficient alpha-L-iduronidase (IDUA) enzyme. Amicus is developing a proprietary human recombinant IDUA (rhIDUA) enzyme co-formulated with a novel pharmacological chaperone as a next-generation therapy for MPS I.
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