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.
Age range
64 Years
Sex
ALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Number of patients with and types of prenatal features of Lysosomal Storage Diseases
Timeframe: 15 years
Number of participants with the presence and levels of glycosaminoglycans (GAGs) in urine.
Timeframe: 15 years
Number of participants that show measured levels of antibodies against the enzyme.
Timeframe: 15 years
Number of participants that show functional cardiac, growth, mobility, and neurocognitive function.
Timeframe: 15 years