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Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who can join

Up to age 64 · All sexes

Inclusion Criteria:

* Patients aged 0-64 with a diagnosis of a lysosomal storage disease
* Pregnant patients whose fetus has a diagnosis of a lysosomal storage disease

Exclusion Criteria:

* There are no current exclusion criteria

About the study

This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.

What is being tested

• There is no intervention (other)

Sponsor: University of California, San Francisco · Participants: 250 · Started: May 31, 2022

Contact the study team

• Billie Lianoglou, MS · Phone: 415-476-2461
• Emma Canepa, MS, CCRP · Phone: 415-476-7255

Official record on ClinicalTrials.gov — NCT05619900

Locations in the U.S.

Conditions

• Mucopolysaccharidosis IV
• Gaucher Disease
• Inborn Errors Metabolism

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.