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Alglucosidase alfa (GZ419829) clinical trials
2 indexed studies · 0 currently recruiting
- Active not recruitingPHASE2
A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa
This study is testing a new version of medicine, avalglucosidase alfa, for children aged 6 months to 17 years with a rare condition called Pompe disease. It's for children who are already on a similar medicine but aren't feeling better or are getting worse. We want to see if the new medicine is safe and works well.
For: Glycogen Storage Disease Type II-Pompe's DiseaseUnited States · France · Japan - Active not recruiting
A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
This study looks at how a treatment called alglucosidase alfa works for babies up to 6 months old with a serious genetic condition called Pompe disease. Researchers want to see if it helps them breathe without a ventilator and how it affects their heart and development.
For: Glycogen Storage Disease Type IIUnited States · Belgium · France