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Article

Pelizaeus-Merzbacher disease: on the cusp of myelin medicine.

Trends in molecular medicine - 1 May 2024

Elitt Matthew S, Tesar Paul J

Abstract excerpt

Pelizaeus-Merzbacher disease (PMD) is caused by mutations in the proteolipid protein 1 (PLP1) gene encoding proteolipid protein (PLP). As a major component of myelin, mutated PLP causes progressive neurodegeneration and eventually death due to severe white matter deficits. Medical care has long been limited to symptomatic treatments, but first-in-class PMD therapies with novel mechanisms now stand poised to enter...

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Identifiers

PMID: 38582621
DOI: 10.1016/j.molmed.2024.03.005
PMCID: PMC11081862

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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