drugset / Trial / NCT00765414
Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.
NaSingle-groupOpen-labelTreatment
Summary
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in a single patient with infantile-onset Pompe disease who were previously treated with rhGAA in a Genzyme study.
Timeline
- Start
- 2003-04
- Primary completion
- 2006-04
- Completion
- 2006-08
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | alglucosidase alfa | Protein / enzyme biologic | 30 mg/kg | — |
| Subject | alglucosidase alfa | Protein / enzyme biologic | 40 mg/kg | — |