Phenotypic spectrum and clinical course of single large-scale mitochondrial DNA deletion disease in the paediatric population: a multicentre study.
Kristoffer BjörkmanJohn VissingElsebet ØstergaardLaurence A BindoffIrenaeus F M de CooMartin EngvallOmar HikmatPirjo IsohanniGittan KollbergChristopher LindbergKari MajamaaKarin NaessJohanna UusimaaMar TuliniusNiklas DarinPublished in: Journal of medical genetics (2021)
Our study provides new insights into the phenotypic spectrum of childhood-onset, LMD-associated syndromes. We found a wider spectrum of more prevalent multisystem involvement compared with previous studies, most likely related to a longer time of follow-up.