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Kuvan, Naglazyme and Aldurazyme Data to Be Presented at the 57th Annual Meeting of the American Society of Human Genetics
Date:10/17/2007

NOVATO, Calif., Oct. 17 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that data from clinical studies of Kuvan(TM) (sapropterin dihydrochloride), Naglazyme(R) (galsulfase), and Aldurazyme(R) (laronidase) will be presented at the 57th Annual Meeting of the American Society of Human Genetics in San Diego, California, October 23-27, 2007.

Data to be presented is summarized below:

Platform Presentation - Session 59 - Therapy for Genetic Disorders

Sapropterin dihydrochloride (sapropterin) increases Phenylalanine (Phe) tolerance in children with phenylketonuria (PKU) maintained on a Phe-restricted diet

Presented by Dr. Barbara Burton of Children's Memorial Hospital, Chicago Saturday, October 27 at 8:30am

-- Double-blind, placebo-controlled Phase 3 study in 90 subjects four to

twelve years of age with a diagnosis of PKU with hyperphenylalaninemia

and controlled on a Phe-restricted diet for more than six months.

-- Patients received sapropterin 20mg/kg/day for eight days. Responders

(greater than or equal to 30% reduction in blood Phe on Day 8) were

randomized 3:1 to sapropterin or placebo for ten weeks.

-- Primary endpoint was daily Phe supplement tolerated during 10 weeks

while maintaining adequate blood Phe control.

-- Mean daily Phe intake (dietary + supplement) increased from 16.8 to

43.8mg/kg/day with sapropterin (p<0.001) and from 16.3 to 23.5mg/kg/day

with placebo (p=0.079).

-- Sapropterin had an acceptable safety profile. The most common adverse

events were headache, abdominal pain, fatigue, and diarrhea.

Phase 3 extension 96-week study for Naglazyme (galsulfase) Enzyme Replacement Therapy (ERT) in MPS VI (Maroteaux-Lamy Syndrome) patients

Presented by Dr. Paul Harmatz of Children's Hospital, Oakland Saturday, October 27 at 8:15am

-- Pivotal, Phase 3 open-label exten
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SOURCE BioMarin Pharmaceutical Inc.
Copyright©2007 PR Newswire.
All rights reserved

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