Registry of Patients Diagnosed With Lysosomal Storage Diseases

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

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

AgeUp to 64

SponsorUniversity of California, San Francisco

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.

Eligibility criteria

Qualifiers

Patients aged 0-64 with a diagnosis of a lysosomal storage disease

Pregnant patients whose fetus has a diagnosis of a lysosomal storage disease

Disqualifiers

There are no current exclusion criteria

Trial design

Treatments tested in this trial

There is no intervention

Treatment groups

250 Participants

are divided into 8 treatment groups

Treatment groups

See each treatment group below.

Sponsors and collaborators

University of California, San Francisco

Lead sponsor

Source ClinicalTrials.gov