New registry aims to unlock secrets of rare lysosomal diseases
NCT ID NCT05619900
First seen Apr 13, 2026 · Last updated May 15, 2026 · Updated 8 times
Summary
This study collects information from up to 250 people with lysosomal storage diseases (like certain forms of MPS, Pompe, Gaucher, and Wolman disease) to learn how these conditions develop and how they appear before birth. Researchers will review medical records and test samples to track symptoms and outcomes. No new treatments are being tested; the goal is to gather knowledge to improve future care.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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University of California San Francisco
RECRUITINGSan Francisco, California, 94143, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Conditions
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