Rocket Pharmaceuticals publishes review of Danon disease cases

Rocket Pharmaceuticals announces the online publication of a comprehensive literature review of published Danon disease cases in the International Journal of Cardiology, co-authored by Eric Adler, M.D., Director of Cardiac Transplant and Mechanical Circulatory Support at UC San Diego Health and Professor of Medicine at University of California San Diego School of Medicine. The manuscript entitled, “Danon Disease: Gender Differences in Presentation and Outcomes” outlines a comprehensive review of 83 published manuscripts describing cases of Danon disease, the target indication for RP-A501. RP-A501 is the company’s adeno-associated viral vector-based gene therapy for the treatment of Danon disease that is in development under a collaboration with the University of California San Diego School of Medicine. The review consisted of 90 male patients and 56 female patients for a total of 146 patients with positive genetic testing for Danon disease or positive muscle biopsy. The results show that despite being an X-linked disease, Danon is as devastating for females as it is males, with 92.5% of overall patients presenting with cardiac abnormalities. The primary outcome of the analysis was defined as a composite outcome of death, heart transplantation, or long-term left ventricular assist device- implantation. The composite outcome occurred in 34.9% of patients, with similar findings seen in males and females. 29 patients received a heart transplantation with complications including allograft rejection leading to re-transplant, muscle weakness and respiratory failure that required mechanical ventilatory support. These findings underscore the limitations of transplantation, which is not considered curative and associated with considerable morbidity and mortality.

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