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The US FDA Lifts the Clinical Hold of Rocket's RP-A501 Clinical Trial to Treat Danon Disease

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The US FDA Lifts the Clinical Hold of Rocket's RP-A501 Clinical Trial to Treat Danon Disease

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  • The FDA decision to lift the hold of P-I clinical trial for RP-A501 is based on the modification of the trial protocol and other supporting documents with revised guidelines for patient selection and management
  • The FDA allows the company to restart the patient enrolment. Additionally- the company is expected to initiate the dosing of the low-dose pediatric patients in Q3’21
  • The P-I trial evaluates the safety and tolerability of RP-A501 therapy (IV infusion) in both pediatric and young adult male patients. The company will report the updated data from the low-dose and higher-dose young adult cohorts in Q4’21

| Ref: Businesswire | Image: Businesswire

Click here to­ read the full press release 

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