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On Monday, Rocket Pharmaceuticals, Inc. (NASDAQ: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 Slides Danon dise
CRANBURY, N.J., November 18, 2024--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, presented long-term safety and efficacy results from the Phase 1 RP-A501 study which showed that RP-A501 was generally well tolerated and all evaluable Danon disease patients demonstrated LAMP2 protein expression at 12 months (sustained up to 60 months) and reduction of l