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Article

Hematopoietic stem cell transplantation in infantile neuronal ceroid lipofuscinosis.

Neurology - 23 Oct 2001

Lönnqvist T, Vanhanen S L, Vettenranta K, Autti T, Rapola J, Santavuori P, Saarinen-Pihkala U M

Abstract excerpt

OBJECTIVE: To study the effect of allogeneic hematopoietic stem cell transplantation (SCT) on the clinical course of infantile neuronal ceroid lipofuscinosis (INCL), a lysosomal storage disease. BACKGROUND: INCL is a progressive encephalopathy with severe neuronal loss, especially in the cerebral and cerebellar cortex and retina. Autofluorescent lipopigments constitute the typical storage material in INCL. The...

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Source

Identifiers

PMID: 11673581
DOI: 10.1212/wnl.57.8.1411

Genes

PPT1
SCT

Provenance

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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