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You are here: Industry Insights > Danon Disease

News List

Rocket Pharmaceuticals Announces Clearance of IND for RP-A501 Gene Therapy for Danon Disease firstwordpharma
January 23, 2019
In parallel with the Phase 1 clinical trial, the Company plans to publish a comprehensive literature review of Danon cases and conduct a retrospective chart review. In addition, the Company will initiate a prospective natural history study with enrollment

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