Rocket Pharmaceuticals, Inc.
(NASDAQ:RCKT) on Monday shared a clinical safety update concerning the first three patients treated in its global, pivotal Phase 2 trial for Danon disease.
Danon disease is a rare, inherited genetic condition characterized by cardiomyopathy (weakening of the heart muscle), skeletal myopathy (muscle weakness) and intellectual disability.
The patients received RP-A501 under a recently modified protocol, demonstrating promising early safety results that pave the way for further clinical progress.
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