New cases that expand the genotypic and phenotypic spectrum of Congenital NAD Deficiency Disorder

This 2021 study by Szot et al. presents new cases that broaden the understanding of Congenital NAD Deficiency Disorder (CNDD), a rare autosomal recessive condition resulting from mutations in the NAXE gene. The authors describe patients exhibiting a spectrum of clinical features, including developmental delay, hypotonia, seizures, and brain abnormalities.