RP-A501
Danon Disease (LAMP2 deficiency)
Phase 2Active - Pivotal Trial Enrolling
Key Facts
Indication
Danon Disease (LAMP2 deficiency)
Phase
Phase 2
Status
Active - Pivotal Trial Enrolling
Company
About Rocket Pharmaceuticals
Rocket Pharmaceuticals is a publicly traded gene therapy company dedicated to developing curative treatments for rare genetic disorders. Its most advanced program targets Danon Disease, a life-threatening cardiac condition, with a Phase 2 pivotal trial underway. The company leverages its expertise in gene therapy platforms to build a pipeline addressing significant unmet medical needs, guided by a patient-centric philosophy and core values of trust, generosity, curiosity, and elevation.
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