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Article

Current international perspectives on hematopoietic stem cell transplantation for inherited metabolic disorders.

Pediatric clinics of North America - 1 Feb 2010

Boelens Jaap J, Prasad Vinod K, Tolar Jakub, Wynn Robert F, Peters Charles

Abstract excerpt

Inherited metabolic disorders (IMD) or inborn errors of metabolism are a diverse group of diseases arising from genetic defects in lysosomal enzymes or peroxisomal function. These diseases are characterized by devastating systemic processes affecting neurologic and cognitive function, growth and development, and cardiopulmonary status. Onset in infancy or early childhood is typically accompanied by rapid...

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PMID: 20307715
DOI: 10.1016/j.pcl.2009.11.004

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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