RCKT: FDA aligns on pivotal Phase 2 trial design for RP-A501 in Danon disease, supporting accelerated approval
FDA confirmed the pivotal Phase 2 trial design for RP-A501 in Danon disease, including a 12-patient efficacy population and 12-month co-primary endpoints to support potential accelerated approval. Initial safety data support continued enrollment under the modified protocol.Original document: Rocket…
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