BioMarin Pharmaceutical and partner Genzyme General have announced newdata showing that Aldurazyme, a recombinant form of the human enzyme alpha-L-iduronidase, is effective for the treatment of mucopolysaccharidosis-I, a crippling and sometimes fatal disease caused by deficiency in the enzyme, for up to two years. The Phase III study's results demonstrated that two years of treatment with Aldurazyme sustained, and in certain measurements improved upon, the positive clinical results recorded at the end of one year of therapy. Clinical and biomedical outcomes included a significantly decreased liver and spleen size, reduced levels of urinary carbohydrates, improvements in physical function and increased height and weight growth rates in the prepubertal patients.
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