Recruiting NCT05619900

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Trial Parameters

Condition Mucopolysaccharidosis I

Sponsor University of California, San Francisco

Study Type OBSERVATIONAL

Phase N/A

Enrollment 250

Sex ALL

Min Age N/A

Max Age 64 Years

Start Date 2022-05-31

Completion 2050-05-31

All Conditions

Mucopolysaccharidosis I Mucopolysaccharidosis II Mucopolysaccharidosis IV A Mucopolysaccharidosis VI Mucopolysaccharidosis VII Pompe Disease Infantile-Onset Neuronopathic Gaucher Disease Wolman Disease

Interventions

There is no intervention

Brief Summary

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.

Eligibility Criteria

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

Related Trials

NCT05619900

Registry of Patients Diagnosed With Lysosomal Storage Diseases

View Trial →

VIEW ON CLINICALTRIALS.GOV ↗ MORE MUCOPOLYSACCHARIDOSIS I TRIALS