Wolman's Disease Completed Phase 2 / 3 Trials for Sebelipase alfa (DB11563)

Also known as: Wolman Disease / Disease, Wolman / Lipidoses NOS / Cerebrotendinous cholesterosis [van Bogaert-Scherer-Epstein]

IndicationStatusPhase
DBCOND0040271 (Wolman's Disease)Completed2 / 3
clinicaltrials.gov IdentifierTitlePurposeDrugs
NCT01371825Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase DeficiencyTreatment