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Data to Be Presented at 58th Annual Meeting of The American Society of Human Genetics
Date:11/6/2008

de in subjects with hyperphenylalaninemia related to primary BH4 deficiency"

767/T - "Differential response to Galsulfase therapy in brothers with mucopolysaccharidosis VI (Maroteaux-Lamy)"

778/T - "Sapropterin (Kuvan) is safe and effective in patients under 4 years of age with PKU"

677/F - "Lysosomal Disease Network, and WORLD Symposium 2009"

787/F - "A follow-up of ERT in two MPS VI patients with poorly engrafted bone marrow transplantation"

805/F - "An improved alpha-glucosidase enzyme for Pompe disease"

About Kuvan

Kuvan (sapropterin dihydrochloride) Tablets is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU). Kuvan is to be used in conjunction with a Phe-restricted diet. The active ingredient in Kuvan, sapropterin dihydrochloride, is the synthetic form of 6R-BH4 (tetrahydrobiopterin), a naturally occurring enzyme cofactor that works in conjunction with phenylalanine hydroxylase (PAH) to metabolize Phe. BioMarin and Merck Serono estimate that Kuvan could be a potential treatment option for approximately 30 percent to 50 percent of the estimated 50,000 identified PKU patients in the developed world.

Kuvan has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMEA). Kuvan has received seven years of market exclusivity in the United States. In November 2007, Merck Serono submitted a Marketing Authorization Application (MAA) to the EMEA for sapropterin dihydrochloride as an oral treatment for patients suffering from HPA due to PKU or BH4 deficiency. If approved in the EU, it will receive 10 years of market exclusivity for this indication.

About Naglazyme

Naglazyme (galsulfase) is the first drug approved to treat MPS VI in the United States and Europe. It is an enzyme replacement therapy, using a recombina
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SOURCE BioMarin Pharmaceutical Inc.
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