New hope for gaucher patients: first human trial of FLT201
NCT ID NCT05324943
First seen Nov 05, 2025 · Last updated May 22, 2026 · Updated 27 times
Summary
This early-stage study tested a new treatment called FLT201 in 6 adults with Gaucher disease type 1. The goal was to see if it is safe and can boost the missing enzyme that causes the disease. Researchers hope this approach may reduce harmful buildup in cells and improve symptoms.
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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.
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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Contacts and locations
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Locations
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Hospital Quironsalud Zaragoza
Zaragoza, Spain
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Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre, Brazil
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Kaiser Permanente
Los Angeles, California, 90027, United States
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Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030-6066, United States
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Rabin Medical Center - PPDS
Petah Tikva, Israel
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Royal Free Hospital
London, United Kingdom
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Salford Royal Hospital
Salford, United Kingdom
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Shaare Zedek Medical Center
Jerusalem, Israel
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SphinCS
Höchheim, Germany
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Tel Aviv Sourasky Medical Center
Tel Aviv, Israel
Conditions
Explore the condition pages connected to this study.