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

and joint abnormalities, which limit mobility and endurance. Malformation of the thorax impairs respiratory function, and odontoid hypoplasia and ligamentous laxity cause cervical spinal instability and potential cord compression. Other symptoms may include hearing loss, corneal clouding, and heart valvular disease. Initial symptoms often become evident in the first five years of life.  Depending on severity of the disease, age of diagnosis will vary.

The rate of incidence of MPS IVA is as yet unconfirmed and varies among different populations but estimates vary between 1 in 200,000 live births and 1 in 250,000 live births. There are several studies that have documented the incidence as high as 1 in 76,000 live births in Northern Ireland. The estimated prevalence is between 1,000 and 1,500 patients in the U.S., EU and Japan and between 1,500 to 2,000 patients in the rest of the world for a total of 2,500 to 3,500 patients. Over 1,000 MPS IVA patients worldwide have been identified through The International Morquio Organization (IMO) survey and the BioMarin MorCAP registry program.

About BioMarinBioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine phosphate), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Synd
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SOURCE BioMarin Pharmaceutical Inc.
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