RWD59 Association of Time of Loss of Ambulation with Severity of Respiratory Symptoms in Patients with Nonsense Mutation Duchenne Muscular Dystrophy (NMDMD)

Abstract

Age at loss of ambulation (AALoA) and respiratory symptoms for nmDMD have been compared between patients receiving ataluren from the STRIDE registry and standard of care (SoC) from the CINRG study. A 4-year delay in loss of ambulation (LoA) was demonstrated for ataluren-treated patients. However, estimating treatment effects on respiratory function is challenging due to data limitations. This study aimed to assess the association between LoA and respiratory function - defined by predicted forced vital capacity (pFVC).