Brain-Targeting gene therapy tested for devastating rare disease
NCT ID NCT03580083
Summary
This early-stage study is testing a one-time gene therapy called RGX-111 for people with MPS I, a rare genetic disorder that affects the brain. The therapy aims to deliver a working copy of a missing gene directly to the central nervous system to help the brain produce a needed enzyme. The main goal is to check if the treatment is safe and tolerated in up to 21 participants, while also looking for early signs of effect on brain development and function.
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 HURLER SYNDROME 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.