P.250Phenotypic variability of GNE myopathy: stratification for clinical trial readiness

GNE myopathy is a disease caused by biallelic GNE mutations, and recently has attracted public attentions since its therapeutic approaches have been promising. Clinical trials require better understanding of the disease, which finally can lead to proper study design and interpretation. This study was aimed to comprehend the progression of GNE myopathy and to stratify the patients for future clinical trials. Forty-four patients with genetically...