Paediatric-onset hereditary spastic paraplegias: a retrospective cohort study.
Silvia SchiavoniCarlotta SpagnoliSusanna RizziGrazia G SalernoDaniele FrattiniFrancesco PisaniCarlo FuscoPublished in: Developmental medicine and child neurology (2020)
A genetic diagnosis of paediatric-onset hereditary spastic paraplegia was achieved in one-third of patients. Pathogenic/likely pathogenic variants in rare genes were found. Genotypic and phenotypic heterogeneity favours targeted panel/whole-exome sequencing for diagnosis.