This study is for adults with late-onset Pompe disease, a rare genetic disorder that affects muscles. Researchers want to learn about the safety and effects of a new drug called DNL952. Pharmacokinetics (PK) is how the body processes a drug, and pharmacodynamics (PD) is how a drug affects the body. Participants will try different doses of DNL952 to see which is safest and most tolerable.
Key points to consider:
- The study involves multiple visits to the research center over a period of time.
- Participants must be able to walk at least 40 meters. Using walking aids is allowed.
- There may be risks involved, like side effects, but these will be monitored closely.
Participants must weigh at least 40 kg and have a confirmed diagnosis of late-onset Pompe disease. They should not have other major health issues unless well-controlled. Some may need to have been on specific enzyme-replacement therapies for the past year. If you rely heavily on machines to help you breathe while awake or have taken an investigational drug recently, you may not be eligible.