Data Monitoring Committee: "An Open-label Ascending Dose Cohort Study to Assess the Safety,Pharmacokinetics, and Preliminary Efficacy of NeoGAA (GZ402666) in Patients with Infantile-onset PompeDisease Treated with Alglucosidase Alfa Who Demonstrate Cli

Medicine & Life Sciences

1. human GAA protein 100%
2. Clinical Trials Data Monitoring Committees 88%
3. Pharmacokinetics 43%
4. Glycogen Storage Disease Type II 41%
5. Cohort Studies 39%
6. Safety 33%