Wolman Disease

• CLINICAL_TRIALS other · 2026-04-08
  Clinical Trial Update: Registry of Patients Diagnosed With Lysosomal Storage Diseases

  This ClinicalTrials.gov registry entry describes ongoing recruitment for a registry of patients diagnosed with various lysosomal storage dis…

• CLINICAL_TRIALS research · 2026-03-06
  Clinical Trial Update: Lysosomal Acid Lipase (LAL) Deficiency Registry

  Clinical trial registry update describing a Lysosomal Acid Lipase (LAL) deficiency registry that is currently recruiting to collect data on …

• CLINICAL_TRIALS trial_result · 2024-05-09
  Clinical Trial Update: Breakfast for Young Women [NA]

  This clinical trial update notes the completion of a University of Aarhus–sponsored study titled 'Breakfast for Young Women,' which investig…

• CLINICAL_TRIALS research · 2016-06-27
  Clinical Trial Update: A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype

  A completed retrospective natural history study of patients with Lysosomal acid lipase deficiency and Wolman disease, documenting clinical f…

• CLINICAL_TRIALS trial_result · 2016-04-18
  Clinical Trial Update: Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency [Phase 2, Phase 3]

  This clinical trial update reports Phase 2/3 results for Sebelipase alfa in children with growth failure due to lysosomal acid lipase defici…

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• CLINICAL_TRIALS research · 2015-07-28
  Clinical Trial Update: Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

  A completed observational study update describing a natural history chart review and longitudinal follow-up for Wolman disease and cholester…