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.
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Contacts and locations
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Locations
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Hospital Sant Joan de Déu
Barcelona, 08950, Spain
Conditions
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