One-Shot gene therapy trial offers new hope for debilitating muscle disease
NCT ID NCT07282847
Summary
This early-stage study is testing a single infusion of an experimental gene therapy called AB-1009 in adults with late-onset Pompe disease. The goal is to see if this one-time treatment is safe and if it can help control the disease, which causes progressive muscle weakness. Participants will be followed for over 5 years to monitor their health and response.
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
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Barrow Neurological Institute
NOT_YET_RECRUITINGPhoenix, Arizona, 85013, United States
Contact
-
Duke University
NOT_YET_RECRUITINGDurham, North Carolina, 27705, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
NYU Langone
NOT_YET_RECRUITINGNew York, New York, 10016, United States
Contact
-
Oregon Health and Science University (OHSU)
NOT_YET_RECRUITINGPortland, Oregon, 97239, United States
Contact
-
Stanford Neuroscience Health Center
NOT_YET_RECRUITINGPalo Alto, California, 94304, United States
Contact
-
University of California, Irvine (UCI)
RECRUITINGIrvine, California, 92697, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
University of Pennsylvania
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact
-
University of Pittsburgh Medical Center (UPMC)
NOT_YET_RECRUITINGPittsburgh, Pennsylvania, 15213, United States
Contact
-
University of Texas Southwest Medical Center
NOT_YET_RECRUITINGDallas, Texas, 75235, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Virginia Commonwealth University (VCU)
NOT_YET_RECRUITINGRichmond, Virginia, 23298, United States
Contact
Conditions
Explore the condition pages connected to this study.