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FDA Aligns on Rocket Pharmaceuticals RP-A501 Phase 2 Trial Design for Danon Disease
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Fusion42 · 17 September 2026 · Fusion42 review
The FDA has provided alignment on the design of Rocket Pharmaceuticals' pivotal Phase 2 trial for RP-A501 targeting Danon disease, including dose recalibration, a 12-patient trial size, and a 12-month co-primary endpoint structure.
This Wire brief sits within Fusion42's coverage of Biotech.
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1 source · 16 Sep 2026
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