Registry of Patients Diagnosed With Lysosomal Storage Diseases
Recruiting
Observational Study
Mucopolysaccharidosis I
Mucopolysaccharidosis II
Mucopolysaccharidosis IV A
Mucopolysaccharidosis VI
Mucopolysaccharidosis VII
Pompe Disease Infantile-Onset
Neuronopathic Gaucher Disease
Wolman Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- Up to 64
- Sex
- Any
- Study type
- Observational
- Participants needed
- 250 (estimated)
- Sponsor
- University of California, San Francisco · Other
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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.
Trial Locations
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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
Contacts