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.
Authors
- Inês Noites (ORCID: https://orcid.org/0000-0003-0600-1429)
- Filipa Alveirinho
- Ana Luísa Rodrigues
- Carla Conceição
- Eulália Viveiros
Institutions
- Hospital de Dona Estefânia (PT)
- Hospital do Divino Espírito Santo (PT)
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