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avalglucosidase alfa clinical trials
2 indexed studies · 0 currently recruiting
- Ongoing, recruitingTherapeutic confirmatory (Phase III)
An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)
This study is testing a new medicine, Nexviadyme, for babies with a rare genetic condition called Pompe disease. It aims to see if the medicine helps them breathe better and grow, and if it's safe, especially for those who haven't tried other treatments.
For: Glycogen storage disease type IISpain · Netherlands · Germany - Active not recruitingPHASE3
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
This study is for babies under one year old newly diagnosed with Infantile-Onset Pompe Disease. It's testing a new medicine, avalglucosidase alfa, to see if it's safe and effective in improving their condition, following them for up to four years.
For: Glycogen Storage Disease Type IIUnited States · Belgium · China