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pro vyhledávání: '"Susan Okazaki"'
Autor:
Michael Diament, Monica Sifuentes, Richard Hoft, Kenneth Huff, Susan Okazaki, Emil D. Kakkis, Robin W. Doroshow, Irwin Walot, Gerald F. Cox, G.R. Mason, Stuart J. Swiedler
Publikováno v:
Molecular genetics and metabolism. 90(2)
Recombinant human α-l-iduronidase (Aldurazyme ® , laronidase) was approved as an enzyme replacement therapy for patients with the lysosomal storage disorder, mucopolysaccharidosis I (MPS I). In order to assess the long-term safety and efficacy of l