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Article

Phenotype assessment for neurodegenerative murine models with ataxia and application to Niemann-Pick disease, type C1.

Biology open - 15 Apr 2022

Yerger Julia, Cougnoux Antony C, Abbott Craig B, Luke Rachel, Clark Tannia S, Cawley Niamh X, Porter Forbes D, Davidson Cristin D

Abstract excerpt

Identifying meaningful predictors of therapeutic efficacy from preclinical studies is challenging. However, clinical manifestations occurring in both patients and mammalian models offer significant translational value. Many neurological disorders, including inherited, metabolic Niemann-Pick disease, type C (NPC), exhibit ataxia. Both individuals with NPC and murine models manifest ataxia, and investigational...

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Identifiers

PMID: 35452076
DOI: 10.1242/bio.059052
PMCID: PMC9096702

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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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