Infantile Neuroaxonal Dystrophy: Clinical Case

Infantile neuroaxonal dystrophy is a rare neurodegenerative disorder caused by mutations in the PLA2G6 gene and is part of the neurodegeneration with brain iron accumulation disease group. We present the case of a three-year-old male with a six-month history of neurodevelopmental regression, including motor and speech regression. Brain magnetic resonance imaging (MRI) revealed characteristic findings such as claval hypertrophy and symmetrical cerebellar atrophy, prompting targeted genetic testing which confirmed a compound heterozygous mutation in the PLA2G6 gene. This case emphasizes the importance of integrating clinical, imaging, and genetic findings for the early diagnosis of infantile neuroaxonal dystrophy, highlighting the critical role of pathognomonic MRI features.

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Publication Details

Journal
Sinapse
Published
2026-01-04
DOI
https://doi.org/10.46531/sinapse/cc/149/2025
Primary Topic
Neurological diseases and metabolism
Type
article
Field-Weighted Citation Impact
0.00
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article

Infantile Neuroaxonal Dystrophy: Clinical Case

Inês Noites, Filipa Alveirinho, Ana Luísa Rodrigues, Carla Conceição et al.
Sinapse
Neurological diseases and metabolism
article

Infantile Neuroaxonal Dystrophy: Clinical Case

Inês Noites, Filipa Alveirinho, Ana Luísa Rodrigues, Carla Conceição, Eulália Viveiros
article en

Abstract

Infantile neuroaxonal dystrophy is a rare neurodegenerative disorder caused by mutations in the PLA2G6 gene and is part of the neurodegeneration with brain iron accumulation disease group. We present the case of a three-year-old male with a six-month history of neurodevelopmental regression, including motor and speech regression. Brain magnetic resonance imaging (MRI) revealed characteristic findings such as claval hypertrophy and symmetrical cerebellar atrophy, prompting targeted genetic testing which confirmed a compound heterozygous mutation in the PLA2G6 gene. This case emphasizes the importance of integrating clinical, imaging, and genetic findings for the early diagnosis of infantile neuroaxonal dystrophy, highlighting the critical role of pathognomonic MRI features.

SinapseVol. 25(4)
Hospital de Dona Estefânia (PT), Hospital do Divino Espírito Santo (PT)
Good health and well-being
Openalex Percentile: Top 98%
Neurological diseases and metabolism
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