Gene Therapy in Heart Failure: A Breakthrough for Danon Disease and Beyond
Dr. Eric Adler recently joined The Heart Failure Beat to discuss the science, story, and significance behind his landmark gene therapy trial for Danon disease—a rare, malignant, and under-recognized cause of inherited cardiomyopathy.
“Essentially, we’re all code,” Adler explains. “And now, we have the tools to fix it.” His team’s work, recently published in the New England Journal of Medicine1 , showed dramatic clinical improvements—up to a 40% reduction in heart mass and a 90% drop in troponins.
Adler walks us through the trial’s origin at the bedside, regulatory hurdles, safety concerns like complement-mediated toxicity, and the promise of evolving delivery platforms. He also speaks candidly about equity, ethics, and the role of patient advocates in driving scientific progress: “Do not mess with the rare disease mom. They’re the reason these cures happen.”
As gene therapy expands to other monogenic diseases, Dr. Adler urges heart failure physicians to lean into genomics. “This is chess, not checkers,” he says. “We need a new generation trained to lead.”
🎧 Listen now and stay at the forefront of transformational heart failure care.