Gene therapy trial hopes to treat rare muscle disease
NCT ID NCT04174105
First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 37 times
Summary
This early-stage study tests a gene therapy called AT845 for adults with late-onset Pompe disease, a condition that weakens muscles. The treatment aims to deliver a working copy of the GAA gene to muscle cells. Eleven participants who have been on standard enzyme replacement therapy for at least two years are enrolled. The study focuses on safety and whether the therapy can improve muscle function.
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 POMPE DISEASE (LATE-ONSET) 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
-
Newcastle Upon Tyne Hospitals Foundation Trust Clinical Research Facility
Newcastle upon Tyne, NE1 4LP, United Kingdom
-
Stanford University
Palo Alto, California, 94304, United States
-
University of California Irvine, Department of Neurology
Orange, California, 92868, United States
-
University of Utah, Division of Medical Genetics
Salt Lake City, Utah, 84108, United States
Conditions
Explore the condition pages connected to this study.