This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).
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Safety and Tolerability over time
Timeframe: Up to month 120
GAA enzymatic activity
Timeframe: Baseline and Week 12
GAA protein expression
Timeframe: Baseline and Week 12