Delandistrogene moxeparvovec-rokl showed significant functional benefits in ambulatory DMD patients compared to external controls in the EMBARK trial. The therapy is the only approved gene therapy for ...
New pharmacokinetic and cardiac biomarker data further demonstrate ifetroban's potential to protect the heart and reduce cardiac damage in DMD patients "Seeing these promising results validates our ...
Delandistrogene moxeparvovec showed significant long-term stabilization or slowed progression in DMD patients over 3 and 5 years compared to external controls. Clinical trials demonstrated ...
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