Rocket Pharmaceuticals Inc.

This is a very early study testing the safety of a single intravenous gene therapy for adults with a specific inherited form of heart muscle weakness (dilated cardiomyopathy). The therapy, called RP-A701, delivers a working copy of a faulty gene (BAG3) directly to the heart. Rese…

This study is testing a one-time gene therapy infusion for males aged 8 and older with Danon Disease, a rare genetic condition that damages the heart. The goal is to see if delivering a corrected gene can improve heart health and slow disease progression. Researchers will measure…

This study aims to learn more about how a specific inherited heart condition (PKP2-ACM) progresses over time in people receiving standard care. Researchers will observe 36 patients who have this genetic variant and an implanted heart device, collecting data on their heart health,…

This study aims to understand how Danon disease, a rare and severe genetic heart condition, progresses over time in patients. Researchers will collect medical information from about 60 male and female patients, both from their past records and by following them forward. The goal …