Neuroimaging Features of Biotinidase Deficiency.
Asthik BiswasCillian McNamaraVykuntaraju K GowdaForam B GalaSniya Valsa SudhakarJai SidpraM S VariPasquale StrianoSusan I BlaserMariasavina SeverinoSpyros BatziosKshitij MankadPublished in: AJNR. American journal of neuroradiology (2023)
Biotinidase deficiency is an autosomal recessive condition caused by pathogenic variants in the BTD gene. Resultant deficiency of free biotin leads to impaired activity of the enzyme carboxylase and related neurologic, dermatologic, and ocular symptoms. Many of these are reversible on treatment, but early recognition and commencement of biotin supplementation are critical. This practice is especially important in countries where routine neonatal screening for biotinidase deficiency is not performed. In this report comprising 14 patients from multiple centers, we demonstrate the MR imaging patterns of this disorder at various age groups. Knowledge of these patterns in the appropriate clinical context will help guide early diagnosis of this treatable metabolic disorder.
Keyphrases
- replacement therapy
- healthcare
- end stage renal disease
- copy number
- newly diagnosed
- primary care
- chronic kidney disease
- prognostic factors
- peritoneal dialysis
- dna methylation
- magnetic resonance
- gene expression
- magnetic resonance imaging
- intellectual disability
- computed tomography
- autism spectrum disorder
- quality improvement
- combination therapy
- contrast enhanced
- optical coherence tomography