An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

drug protocol number
LAL-CL03
drug sponsor
Synageva
drug study
Sebelipase alfa (SBC-102)
drug trial site
KFSH & RC-R
drug status
Completed
drug phase