Gene therapy trial tests new approach for rare genetic disorder
NCT ID NCT05324943
Summary
This first-in-human study tested the safety and early effects of a new gene therapy called FLT201 in adults with Gaucher disease type 1. The therapy aims to boost a missing enzyme to help the body break down harmful substances that build up in cells. Six adults who were already on standard treatment participated to see if FLT201 could safely increase enzyme activity and potentially improve their condition.
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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.