Rocket Pharmaceuticals Announces Completion of Enrollment in Phase 2 Pivotal Trial of RP-A501 for the Treatment of Danon Disease
Rocket Pharmaceuticals, Inc. (RCKT)
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Source: Business Wire
CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that all patients have been enrolled in the global, pivotal Phase 2 clinical trial evaluating RP-A501 to treat male patients with Danon disease.After the two-patient safety run-in, followed by harmonized global site activations, the remaining 10 patients were enrolled across the United States (U.S.) and European Union within three months. Given the prevalence of Danon disease across regions, the Company plans to pursue regulatory filings concomitantly in the U.S. and ex-U.S.“From a clinical perspective, the important thing is that we are moving closer to the goal of having a treatment for patients with Danon disease,” said Barry H. Greenberg, MD, FHFSA, Director of the Advanced Heart Failure Treatment Program and Distinguished Professor of Med
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News
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RCKT
Earnings
- 8/5/24 - In-Line
RCKT
Sec Filings
- 11/4/24 - Form 4
- 11/4/24 - Form 4
- 10/31/24 - Form 144
- RCKT's page on the SEC website