Newborn Screening and Early Cord Blood Transplant for Mucopolysaccharidosis

This case series describes the presymptomatic treatment of infants with mucopolysaccharidoses detected by newborn screening using enzyme replacement therapy and reduced-intensity conditioning cord blood transplant, combined with intracerebroventricular enzyme replacement therapy.

Corresponding Author: Hirotoshi Sakaguchi, MD, PhD, Children’s Cancer Center, National Center for Child Health and Development, 2-10-1, Okura, Setagaya-ku, Tokyo 157-8535, Japan (sakaguchi-hi@ncchd.go.jp).

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