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An Open-label, Single Arm, Dose-Escalation Clinical Study to Evaluating the Safety, Tolerability and Preliminary Efficacy of a Single Intracerebroventricular Injection of RB001 for the Treatment of SHANK3-related Phelan McDermid Syndrome

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Trial Profile

An Open-label, Single Arm, Dose-Escalation Clinical Study to Evaluating the Safety, Tolerability and Preliminary Efficacy of a Single Intracerebroventricular Injection of RB001 for the Treatment of SHANK3-related Phelan McDermid Syndrome

Status: Not yet recruiting
Phase of Trial: Phase I

Latest Information Update: 17 Jun 2025

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At a glance

  • Drugs RB 001 (Primary)
  • Indications Telomeric 22q13 Monosomy Syndrome
  • Focus Adverse reactions; First in man

Most Recent Events

  • 17 Jun 2025 New trial record

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