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Case series shows presymptomatic treatment improves outcomes in newborns with mucopolysaccharidosis using enzyme replacement therapy.
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.
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Sakaguchi et al. (2026) studied this question.
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