Real-world experience with gene therapy in Duchenne muscular dystrophy center readiness and patients safety: report from Qatar

This study has described eight DMD patients, who successfully have received Delandistrogene Moxeparvovec (Elevidys) in a specialized center in Qatar.

This study explored the physiological responses to gene therapy in a cohort of DMD patients, assessing the impact on biomarker trends over a 30-week period post-infusion. All patients received comprehensive pre-infusion assessment including antibody screening, corticosteroids dosing, antihistamines, antiemetics, and supportive physiotherapy and cardiac assessment.

Our observations of biomarker…