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Treatment
Cipaglucosidase alfa clinical trials
2 indexed studies · 2 currently recruiting
- Recruiting
A Global Prospective Observational Registry of Patients With Pompe Disease
This study is gathering information on people with Pompe disease, both those receiving treatment and those not yet treated. It aims to understand how treatments affect health and daily life over time, and what happens when the disease is untreated.
For: Pompe DiseaseUnited States · Austria · Belgium - RecruitingPHASE3
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
This study looks at a new treatment called cipaglucosidase alfa/miglustat for children with a rare genetic condition called Infantile Onset Pompe Disease (IOPD). It checks if the treatment is safe and works, and how the body handles it. It includes children who have and haven't had previous treatments.
For: Glycogen Storage Disease Type II Infantile OnsetUnited States · Germany · Italy