Gene therapy trial aims to bypass immune barriers in devastating muscle disease
NCT ID NCT06241950
Summary
This study tested a one-time gene therapy (SRP-9001) for boys with Duchenne muscular dystrophy (DMD) who had pre-existing antibodies that would normally block the treatment. Researchers first gave a drug called imlifidase to temporarily lower these antibodies, then administered the gene therapy. The main goals were to check the safety of this combined approach and to see if the therapy successfully delivered the needed gene to muscle tissue.
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.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
-
Hospital Sant Joan de Déu
Barcelona, 08950, Spain
Conditions
Explore the condition pages connected to this study.