Prospective 5‐year natural history study of infantile <scp> <i>PLA2G6</i> </scp> ‐associated neurodegeneration

In this first prospective natural history study of infantile PLAN, we characterize developmental and disease milestones, and longitudinal associations, with quality of life metrics. These data will provide valuable disease norms for use as clinical references and in the design of interventional trials.

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Publication Details

Journal
Developmental Medicine & Child Neurology
Published
2025-08-10
DOI
https://doi.org/10.1111/dmcn.16464
Primary Topic
Neurological diseases and metabolism
Type
article
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article

Prospective 5‐year natural history study of infantile PLA2G6 ‐associated neurodegeneration

Penelope Hogarth, Jenny L. Wilson, Allison Gregory, Kim Anderson et al.
1 links
Developmental Medicine & Child Neurology
Neurological diseases and metabolism
article

Prospective 5‐year natural history study of infantile PLA2G6 ‐associated neurodegeneration

Penelope Hogarth, Jenny L. Wilson, Allison Gregory, Kim Anderson, Robin L. Baudier, Hannah Loftus, S. J. Hayflick
article en

Abstract

In this first prospective natural history study of infantile PLAN, we characterize developmental and disease milestones, and longitudinal associations, with quality of life metrics. These data will provide valuable disease norms for use as clinical references and in the design of interventional trials.

Developmental Medicine & Child NeurologyVol. 68(4)
Oregon Health & Science University (US)
Openalex Percentile: Top 74%
Neurological diseases and metabolism
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