New gene therapy aims to fix bone marrow in rare telomere disease
NCT ID NCT04211714
First seen Nov 01, 2025 · Last updated Apr 30, 2026 · Updated 30 times
Summary
This early-phase study tests a new treatment called EXG34217 in 12 people aged 12 and older who have bone marrow failure due to telomere biology disorders. The main goal is to see if the treatment is safe and tolerable. Researchers will also check if it improves blood cell counts and telomere length.
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 TELOMERE SHORTENING 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
-
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
Conditions
Explore the condition pages connected to this study.