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Article

Emerging treatments for pediatric leukodystrophies.

Pediatric clinics of North America - 1 Jun 2015

Helman Guy, Van Haren Keith, Escolar Maria L, Vanderver Adeline

Abstract excerpt

The leukodystrophies are a heterogeneous group of inherited disorders with broad clinical manifestations and variable pathologic mechanisms. Improved diagnostic methods have allowed identification of the underlying cause of these diseases, facilitating identification of their pathologic mechanisms. Clinicians are now able to prioritize treatment strategies and advance research in therapies for specific disorders....

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Identifiers

PMID: 26022168
DOI: 10.1016/j.pcl.2015.03.006
PMCID: PMC5712822

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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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