Enzyme replacement therapy in patients who have mucopolysaccharidosis i and are younger than 5 years: Results of a multinational study of recombinant human α-L-iduronidase (laronidase)

J. Edmond Wraith, Michael Beck, Roderick Lane, Ans Van Der Ploeg, Elsa Shapiro, Yong Xue, Emil D. Kakkis, Nathalie Guffon

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Medicine & Life Sciences