Gene therapy for gaucher disease: 5-year safety check begins
NCT ID NCT06545136
First seen Nov 05, 2025 · Last updated May 07, 2026 · Updated 22 times
Summary
This study follows 75 people with Gaucher disease type 1 who already received an experimental gene therapy called FLT201. Researchers will monitor them for 5 years to see if the treatment remains safe and if its effects last. The goal is to understand long-term risks and benefits of this one-time gene therapy.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for GAUCHER DISEASE, TYPE 1 are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Hospital Quironsalud Zaragoza
Zaragoza, Spain
-
Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre, Brazil
-
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030-6066, United States
-
Salford Royal Hospital
Salford, United Kingdom
Conditions
Explore the condition pages connected to this study.