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Connective Tissue Diseases

July 8, 2002

Results from a 24-week, open-label extension of a phase I trial indicate that both dose levels (0.2 mg/kg and 1.0 mg/kg) of BioMarin Pharmaceutical's Aryplase continue to be well tolerated by the five subjects who have received treatment for a total of 48 weeks. Aryplase is an enzyme replacement therapy being developed for the treatment of mucopolysaccharidosis VI. In addition to the positive safety results, the 1.0 mg/kg dose continued to produce a greater sustained reduction than the 0.2 mg/kg dose in the excretion of urinary glycosaminoglycans.