RCKT Completes Enrollment in Rare Heart Disease Study, Stock Rises

Rocket Pharmaceuticals, Inc. (RCKT) announced that it has completed patient enrollment in a pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating male patients with Danon disease. Danon disease is a rare X-linked inherited disorder caused by mutations in the LAMP2 gene. Per the company, an estimated 15,000 to 30,000 patients suffer from Danon disease in the United States and Europe. Currently, the only available treatment for the disease is cardiac transplantati ...