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BioMarin Initiates Pivotal Phase 3 Trial for GALNS for the Treatment of MPS IVA
Date:2/1/2011

rome (LEMS). Other product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, and BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking StatementThis press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the development of its program for MPS IVA, and particularly the timing and conduct of clinical trials related thereto, expectations regarding other clinical and preclinical programs, and the potential market for GALNS, if approved. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the results of current and planned pre-clinical trials related to BioMarin's development programs and particularly the enzyme replacement therapy for MPS IVA; the content and timing of decisions by the U.S. Food and Drug Administration, EMEA and other regulatory agencies, particularly with respect to the enzyme replacement therapy for MPS IVA, the actual number of MPS IVA patients in the developed world; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, includi
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