Gene therapy for Danon disease successful

Gene therapy for Danon disease successful

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medicine

Wednesday, November 20, 2024

San Diego – A gene therapy that endows muscle cells with an intact version of the LAMP2 gene has improved cardiac symptoms in a Phase 1 trial in patients with Danon disease, a rare X-linked disorder that affects childhood male patients leads to heart failure. Results were presented at the annual meeting of the American Heart Association and published in the New England Journal of Medicine (2024;…

#Gene #therapy #Danon #disease #successful

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