Gene therapy shows promise for kids with SMA after other drugs fail
NCT ID NCT05386680
First seen Nov 01, 2025 · Last updated May 07, 2026 · Updated 29 times
Summary
This study tested a one-time gene therapy called OAV101 in 27 children aged 2 to 18 with spinal muscular atrophy (SMA) who had stopped taking other SMA medicines. The goal was to see if the treatment was safe and could help maintain or improve muscle function. Researchers monitored side effects and measured motor skills over a year.
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 SPINAL MUSCULAR ATROPHY 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
-
Boston Childrens Hospital
Boston, Massachusetts, 02215, United States
-
Child Hosp Of The Kings Daughters
Norfolk, Virginia, 23507, United States
-
Novartis Investigative Site
Parkville, Victoria, 3052, Australia
-
Novartis Investigative Site
Leuven, 3000, Belgium
-
Novartis Investigative Site
Montreal, Quebec, H4A 3J1, Canada
-
Novartis Investigative Site
Bron, 69677, France
-
Novartis Investigative Site
Toulouse, 31059, France
-
Novartis Investigative Site
Roma, RM, 00168, Italy
-
Novartis Investigative Site
Kurume, Fukuoka, 830-0011, Japan
-
Novartis Investigative Site
Shinjuku Ku, Tokyo, 162 8666, Japan
-
Novartis Investigative Site
Utrecht, 3584, Netherlands
-
Novartis Investigative Site
Barcelona, Catalonia, 08035, Spain
-
University of Wisconsin Madison Medical School
Madison, Wisconsin, 53792-7375, United States
Conditions
Explore the condition pages connected to this study.