Gene therapy trial halted for boys with rare muscle disease
NCT ID NCT06241950
Summary
This study tested a two-step treatment for Duchenne muscular dystrophy (DMD). First, a drug (imlifidase) was given to temporarily lower antibodies that might block the gene therapy. Then, the gene therapy (SRP-9001) was given to try to help muscles produce a needed protein. The small, early-stage trial was terminated and aimed to see if this approach was safe and if the therapy worked in the muscle.
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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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