PMPCA-Related Encephalopathy: Novel Variants, Phenotype Extension, and Mitochondrial Morphology.
Vibhuti RambaniMiriam KolnikovaMichal CagalinecMartina ŠkopkováDaniela GasperikovaPublished in: Neurology. Genetics (2023)
-related disease with a severe intermediate phenotype. Moreover, we extend the phenotype to Leigh-like white matter changes that have not been described in previously reported cases.