Blood filtering paves way for gene therapy in boys with rare muscle disease
NCT ID NCT06597656
Summary
This early-stage study tested a one-time gene therapy for Duchenne muscular dystrophy (DMD) in a very specific group: three boys who already had antibodies that might block the treatment. To try and get around this, doctors first used a blood-filtering procedure called plasmapheresis. The main goals were to see if this approach was safe and if the therapy could successfully deliver a working gene to produce a needed muscle protein.
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 DUCHENNE 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
Locations
-
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
-
University of Florida, College of Medicine
Gainesville, Florida, 32610, United States
-
Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.