Rocket Pharmaceuticals achieves a significant milestone in their RP-A501 therapy for Danon Disease, strengthening their position in the gene therapy industry. RP-A501 aims to restore normal cardiac ...
All six evaluable Phase 1 patients with Danon disease are alive and transplant-free up to age 25 years RP-A501 demonstrated safety and meaningful efficacy; all evaluable patients show cardiac LAMP2 ...
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