Gene therapy watch: 21 patients tracked for years after single treatment
NCT ID NCT06103487
First seen May 04, 2026 · Last updated May 15, 2026 · Updated 1 time
Summary
This study follows 21 people with MPS I who already received RGX-111 gene therapy in an earlier trial. Researchers will monitor them for years to check for side effects and see if the treatment helps with thinking, learning, and daily skills. The goal is to understand the long-term safety 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 MUCOPOLYSACCHARIDOSIS I 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
-
Children's Hospital of Orange County
Orange, California, 92868, United States
-
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903, Brazil
Conditions
Explore the condition pages connected to this study.