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Recruiting
NCT05619900
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Conditions: Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
Sex: All
Ages: N/A – 64 Years
Enrollment: 250
Sponsor: University of California, San Francisco
Location: United States
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 diseasePregnant patients whose fetus has a diagnosis of a lysosomal storage diseaseExclusion Criteria:There are no current exclusion criteria
Source: ClinicalTrials.gov (NCT05619900). StuddyBuddy aggregates publicly available trial information.
