Gene therapy for rare blindness passes 5-year safety check
NCT ID NCT03597399
First seen Sep 30, 2025 · Last updated May 15, 2026 · Updated 20 times
Summary
This study followed 87 people who had already received Luxturna gene therapy for a rare inherited eye condition that can cause blindness. The goal was to watch for any side effects over 5 years after treatment. No new treatment was given; researchers simply collected safety information to better understand the long-term risks.
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 CONFIRMED BIALLELIC RPE65 MUTATION-ASSOCIATED RETINAL DYSTROPHY 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
-
Bascom Palmer Eye Institute
Miami, Florida, 33136, United States
-
Casey Eye Institute
Portland, Oregon, 97239, United States
-
Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
-
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104, United States
-
Cincinnati Eye Institute
Cincinnati, Ohio, 45242, United States
-
Cullen Eye Institute
Houston, Texas, 77030, United States
-
Kellogg Eye Center
Ann Arbor, Michigan, 48105, United States
-
Massachusetts Eye and Ear Institute
Boston, Massachusetts, 02114, United States
-
Scheie Eye Institute
Philadelphia, Pennsylvania, 19104, United States
-
University of Iowa Hospitals & Clinics
Iowa City, Iowa, 52242, United States
Conditions
Explore the condition pages connected to this study.