Hematopoietic stem cell transplantation in mucopolysaccharidosis type IIIA: A case description and comparison with a genotype-matched control group

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Abstract
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Funding Information
  • Amicus
  • Biomarin
  • Sanofi Genzyme
  • Shire
  • Actelion
  • Chiesi Farmaceutici S.p.A.

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