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FDA Lifts Rocket Pharmaceuticals Study Hold For Rare Disease Gene Therapy

Rocket Pharmaceuticals Inc. RCKT stock is trading higher on Wednesday after the U.S. Food and Drug Administration (FDA) lifted the clinical hold on its pivotal Phase 2 trial of RP-A501 for Danon disease. The hold was lifted in under three months. 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. For the unversed, in May, the company reported a patient death in t ...