Rocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments: Analyst

On Monday, Rocket Pharmaceuticals, Inc. RCKT presented long-term safety and efficacy results from the Phase 1 study of RP-A501 in male patients with Danon disease.Danon disease is a rare X-linked dominant genetic disorder that manifests with the clinical triad of cardiomyopathy (stiff heart muscles), skeletal myopathy (weakness), and intellectual disability.Also Read: FDA Rejects Rocket Pharmaceuticals' Gene Therapy For Rare Immune Disorder, Asks For More Data, Stock SlidesDanon disease is caused by mutatio ...