S-606001 clinical trials

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

This study is testing a new medicine, S-606001, for people with late-onset Pompe disease who are already on treatment. It looks at how safe the new medicine is, how it works in the body, and if it helps improve symptoms.