Gene therapy trial hopes to slow rare muscle disease
NCT ID NCT05230459
First seen Oct 31, 2025 · Last updated May 13, 2026 · Updated 31 times
Summary
This study tests a single dose of a gene therapy called AB-1003 in 10 adults with a rare genetic muscle disease (LGMD2I/R9) that causes progressive weakness. The main goal is to check safety, not yet to prove it works. Participants must be able to walk 10 meters in under 30 seconds.
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 LIMB GIRDLE MUSCULAR DYSTROPHY 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: •••••@•••••
-
Contact
Locations
-
Kennedy Krieger Institute
RECRUITINGBaltimore, Maryland, 21205, United States
Contact
Contact Email: •••••@•••••
-
University of California - Irvine
RECRUITINGIrvine, California, 92697, United States
Contact
Contact Email: •••••@•••••
-
University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
Contact Email: •••••@•••••
Contact
-
University of Kansas Medical Center
RECRUITINGKansas City, Kansas, 66160, United States
Contact Email: •••••@•••••
Contact
-
University of Washington Medical Center
RECRUITINGSeattle, Washington, 98195, United States
Contact
Contact Email: •••••@•••••
-
VCU
RECRUITINGRichmond, Virginia, 23298, United States
Contact Email: •••••@•••••
Contact
Conditions
Explore the condition pages connected to this study.