The New England Journal of Medicine (NEJM) is an English-language peer-reviewed medical journal published by the Massachusetts Medical Society. It describes itself as the oldest continuously ...
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Promising results from phase 1 gene therapy trial for Danon diseaseMore information: Barry Greenberg et al, Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease, New England Journal of Medicine (2024). DOI: 10.1056/NEJMoa2412392 ...
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Gene Therapy Promising for Rare Genetic CardiomyopathyCHICAGO -- Gene therapy for Danon disease, a rare inherited cause ... group wrote in a paper simultaneously published in the New England Journal of Medicine. "There really is no treatment at ...
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