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An open-label study of pharmacokinetics, safety, efficacy, and pharmacodynamics of ATB200/AT2221 in pediatric subjects aged 12 to < 18 years with late-onset Pompe Disease (20192533)
The purpose of this study is to find out if an investigational new drug combination called cipaglucosidase alfa/miglustat can safely help children with a rare…
- Ages
- 0 Years - 17 Years
- Sexes
- All