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Article

Rational Design of Novel Therapies for Pantothenate Kinase-Associated Neurodegeneration.

Movement disorders : official journal of the Movement Disorder Society - 1 Sept 2021

Thakur Nivedita, Klopstock Thomas, Jackowski Suzanne, Kuscer Enej, Tricta Fernando, Videnovic Aleksandar, Jinnah Hyder A

Abstract excerpt

BACKGROUND: This review highlights the recent scientific advances that have enabled rational design of novel clinical trials for pantothenate kinase-associated neurodegeneration (PKAN), a rare autosomal recessive neurogenetic disorder associated with progressive neurodegenerative changes and functional impairment. PKAN is caused by genetic variants in the PANK2 gene that result in dysfunction in pantothenate...

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Source

Identifiers

PMID: 34002881
DOI: 10.1002/mds.28642

Genes

PANK2

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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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