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RECRUITINGOBSERVATIONAL

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - Patients aged 0-64 with a diagnosis of a lysosomal storage disease - Pregnant patients whose fetus has a diagnosis of a lysosomal storage disease Who Should NOT Join This Trial: - There are no current exclusion criteria Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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

Treatments Being Tested

OTHER

There is no intervention

This is an observational study. There is no intervention. The purpose of the project is to create a database of patients diagnosed either prenatally or after birth with a lysosomal storage disease. The database will be utilized to assess patient outcomes, build on existing clinical management, improve medical decision making, and improve quality of care.

Locations (1)

University of California San Francisco
San Francisco, California, United States