PCR67 Association of Walking Distance with Quality of Life and Other Patient-Reported Outcomes in Pompe Disease

Abstract

Timed walking tests in clinical studies of certain rare muscular disorders are important efficacy measures appraised in health technology assessments (HTA). We explored whether the six-minute walk distance (6MWD) was associated with various patient-reported outcomes (PROs), including quality of life, in Pompe Disease (PD) – a rare muscular disorder.