Natural History Studies: Danon Disease: Revision history

Jump to navigation Jump to search

Diff selection: Mark the radio buttons of the revisions to compare and hit enter or the button at the bottom.
Legend: (cur) = difference with latest revision, (prev) = difference with preceding revision, m = minor edit.

4 September 2025

  • curprev 03:0203:02, 4 September 2025TerriePeralta33 talk contribs 5,480 bytes +5,480 Created page with "<br>RP-A501 is being developed for Danon Disease, a rare genetic disorder characterized by thickening and weakening of the heart muscle, often resulting in heart failure, and for male patients, frequent death during adolescence or early adulthood. Rocket is the first company demonstrating safety and efficacy data in clinical studies for gene therapy targeting the heart. An open-label, global Phase 2 pivotal clinical trial of RP-A501 for the treatment of Danon Disease has..."