Gait analysis reveals new outcome measures for monitoring disease progression in individuals with late-onset Pompe disease

The Pompe Mobility-Derived Progression Index (Pompe-MDPI) was developed and validated as a sensitive biomarker of disease progression. Longitudinal analysis demonstrated that Pompe-MDPI captured gait deterioration over one year, outperforming traditional measures like the six-minute walk test in sensitivity. These findings support the use of wearable gait analysis as a clinically meaningful, scalable tool for monitoring motor function in LOPD, with implications for both patient care and therapeutic trials.