RecruitingNCT05619900

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Sponsor

University of California, San Francisco

Enrollment

250 participants

Start Date

May 31, 2022

Study Type

OBSERVATIONAL

Conditions

Mucopolysaccharidosis I Mucopolysaccharidosis II Mucopolysaccharidosis VI Mucopolysaccharidosis VII Mucopolysaccharidosis IV A

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

Max Age: 64 Years

Inclusion Criteria2

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

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Interventions

OTHERThere 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.