Scientists track patients for 5 years after pioneering gene therapy
NCT ID NCT06545136
Summary
This study is following 75 adults with Gaucher disease type 1 for five years after they received an experimental gene therapy called FLT201. The main goal is to monitor their long-term safety and see how well the treatment's effects last over time. It is a follow-up to an earlier trial, so it is not recruiting new patients who haven't already received the therapy.
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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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Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030-6066, United States
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Salford Royal Hospital
Salford, United Kingdom
Conditions
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