Longitudinal Motor Function Changes in Adults With Late-Onset Pompe Disease

Our results found significant deterioration in motor parameters in adults with LOPD. Hip muscle deterioration contributes directly to gait and stability decline while plantar flexors play a pivotal role in maintaining cautious gait and balance. The aim of this study was to identify sensitive-to-change parameters and MCID thresholds to provide clinicians with meaningful benchmarks to guide evaluations and interventions. Hip extensor and abductor strength (dynamometer) and walking speed emerged as the most responsive parameters, recommended for monitoring disease progression, optimizing rehabilitation, and assessing emerging treatments. Further studies are needed to confirm these findings in broader LOPD populations and evaluate treatment effects over longer periods.