Targeted Therapies for Leigh Syndrome: Systematic Review and Steps Towards a 'Treatabolome'.

ABSTRACT:

Background

Leigh syndrome (LS) is the most frequent paediatric clinical presentation of mitochondrial disease. The clinical phenotype of LS is highly heterogeneous. Though historically the treatment for LS is largely supportive, new treatments are on the horizon. Due to the rarity of LS, large-scale interventional studies are scarce, limiting dissemination of information of therapeutic options to the wider scientific and clinical community.

Objective

We conducted a systematic review of pharmacological therapies of LS following the guidelines for FAIR-compliant datasets.

Methods

We searched for interventional studies within Clincialtrials.gov and European Clinical trials databases. Randomised controlled trials, observational studies, case reports and case series formed

SUBMITTER: Tiet MY 

PROVIDER: S-EPMC8673543 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature