Wolman's Disease (DBCOND0040271)
Identifiers
- Synonyms
- Wolman Disease / Wolman's disease / Other lipid storage disorders / Wolman's disease (disorder) / Lysosomal acid lipase deficiency / Lysosomal acid lipase deficiency (disorder)
Associated Data
- Indicated Drugs and Targets
- Not Available
- Clinical Trials
Identifier Title Drug(s) Purpose Phase Status NCT00668564 Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism treatment 2 terminated NCT04532047 In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases treatment 1 recruiting NCT01358370 A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype No drug interventions Not Available Not Available completed NCT06287658 The Effect of Kegel Exercise and Ba Duan Jin Applications on Premenopausal Women With Urinary Incontinence No drug interventions treatment Not Available recruiting NCT01371825 Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency treatment 2 / 3 completed NCT00383448 HSCT for High Risk Inherited Inborn Errors treatment 2 completed NCT01633489 Lysosomal Acid Lipase (LAL) Deficiency Registry No drug interventions Not Available Not Available recruiting NCT01586455 Human Placental-Derived Stem Cell Transplantation No drug interventions treatment 1 completed NCT04792671 Prevalence and Risk Factors of Women Mental Health Disorders No drug interventions Not Available Not Available unknown_status NCT00005900 Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation No drug interventions Not Available Not Available unknown_status NCT05619900 Registry of Patients Diagnosed With Lysosomal Storage Diseases No drug interventions Not Available Not Available recruiting NCT04652713 Breakfast for Young Women No drug interventions prevention Not Available completed NCT01884220 Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up No drug interventions Not Available Not Available completed NCT00176904 Stem Cell Transplant for Inborn Errors of Metabolism treatment 2 / 3 completed NCT01473875 Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102 treatment 2 / 3 terminated