Wolman's Disease Completed Phase 2 / 3 Trials for Sebelipase alfa (DB11563)
Also known as: Wolman Disease / Other lipid storage disorders / Wolman's disease (disorder) / Lysosomal acid lipase deficiency / Lysosomal acid lipase deficiency (disorder)
| Indication | Status | Phase |
|---|---|---|
| DBCOND0040271 (Wolman's Disease) | Completed | 2 / 3 |
| clinicaltrials.gov Identifier | Title | Purpose | Drugs |
|---|---|---|---|
| NCT01371825 | Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency | Treatment |