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BioMarin Announces Program for ERT for Treatment of MPS IVA - Morquio A Syndrome
Date:6/5/2008

bout MPS IVA

Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome) is a disease characterized by deficient activity of N-acetylgalactosamine-6- sulfatase (GALNS) causing excessive lysosomal storage of keratan sulfate (KS). This excessive storage causes a systemic skeletal dysplasia, short stature, 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 potentially 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, but estimates vary between 1 in 200,000 live births to 1 in 300,000 live births. Approximately 370 patients worldwide are currently registered in The International Morquio Organization (IMO) survey. An estimated 1,100 patients in the developed world have MPS VI and an estimated 3,000 patients in the developed world have MPS I.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises three approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include 6R-BH4 for cardiovascular in
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SOURCE BioMarin Pharmaceutical Inc.
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