Phase 2 randomized double-blind study of S-606001 added to enzyme replacement therapy in adults with late-onset Pompe disease

Phase 2, randomised, double-blind trial in Pompe disease comparing S-606001 add-on to enzyme replacement therapy with Placebo add-on to enzyme replacement therapy (2 arms). Primary endpoint: Change from baseline in percent predicted forced vital capacity at 52 weeks. Planned start Jun 2026; status: not yet recruiting.