For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety an…

MPS IMPS IIMPS IVA+6 more

~Estimated

~$10 – $5,625

University of California, San FranciscoNCT04532047

Registry of Patients Diagnosed With Lysosomal Storage Diseases

This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the dis…

Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more

~Estimated

~$10 – $675

University of California, San FranciscoNCT05619900

An Observational Disease and Clinical Outcomes Registry of Patients With Lysosomal Acid Lipase (LAL) Deficiency

This is an observational, multi-center, international disease registry designed to collect longitudinal data and create a knowledge base that will be utilized …

Lysosomal Acid Lipase DeficiencyCholesterol Ester Storage DiseaseWolman Disease+4 more

~Estimated

~$10 – $1,050

Alexion Pharmaceuticals, Inc.NCT01633489