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BioMarin Announces Initiation of Clinical Assessment Program for Morquio A Syndrome
Date:11/3/2008

urton, M.D., Director, MPS/ML Treatment Program, Children's Memorial Hospital."

BioMarin has developed and manufactures two FDA-approved enzyme replacement therapies, one for the treatment of MPS I and one for the treatment of MPS VI. Naglazyme(R) (galsulfase) for MPS VI is wholly developed and commercialized by BioMarin. Aldurazyme(R) (laronidase) for MPS I is manufactured by BioMarin and marketed by Genzyme Corporation.

About 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 as well as macrophage storage in the lung likely impairs respiratory function and contributes to sinopulmonary infections. Odontoid hypoplasia and ligamentous laxity can commonly cause cervical spinal instability and potentially spinal cord compression. Other symptoms may include recurrent infections, 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. Many patients end up wheelchair dependent in their second decade of life and undergo numerous surgeries to manage their disease.

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 400 patients worldwide are currently registered in a public registry, based on their publications. The prevalence of patients with MPS IVA appears substantially higher than that with MPS VI based on published reports.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditio
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SOURCE BioMarin Pharmaceutical Inc.
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