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.
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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