Registry following people with rare lysosomal storage diseases over time
Records the natural course of lysosomal storage diseases and outcomes of fetal therapies.
Plain-language title generated from the registry record
Registry title: Registry of Patients Diagnosed With Lysosomal Storage Diseases
Key facts
- Where
- San Francisco, United StatesSee the site
Where in United States
- University of California San FranciscoSan Francisco, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- University of California, San Francisco
- Trial code
- NCT05619900
- Record updated
- 8 April 2026
- Original description
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.Read full original descriptionShow less
Registry of Patients Diagnosed With Lysosomal Storage Diseases
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.
Inclusion: 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: There are no current exclusion criteria.