Wolman Disease Clinical Trials

A listing of Wolman Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 3 clinical trials
In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs). (IUERT)

The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

replacement therapy
  • 11 views
  • 17 Oct, 2022
  • 1 location
Lysosomal Acid Lipase (LAL) Deficiency Registry

of patients with LAL Deficiency. Participation in the Registry by both physicians and patients is voluntary.

deficiency
lipase
  • 283 views
  • 04 Oct, 2022
  • 28 locations
Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects)

responsible for hydrolyzing cholesterol esters and triglycerides that are delivered to lysosomes. Mutations in LIPA that completely inactivate LAL are the molecular cause of Wolman disease, a rapidly lethal

protease
low density lipoprotein
lipa gene
storage disease
familial hypercholesterolemia
  • 3 views
  • 04 Oct, 2022
  • 4 locations