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A global, open-label, multi-center, dose escalation study to evaluate the safety, tolerability of AAV based therapeutics in subjects with Charcot-Marie-Tooth Neuropathy Type 2A (CMT2A)

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Trial Profile

A global, open-label, multi-center, dose escalation study to evaluate the safety, tolerability of AAV based therapeutics in subjects with Charcot-Marie-Tooth Neuropathy Type 2A (CMT2A)

Status: Planning
Phase of Trial: Phase I

Latest Information Update: 12 Sep 2019

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At a glance

  • Drugs PBFT 02 (Primary)
  • Indications Charcot-Marie-Tooth disease
  • Focus Adverse reactions

Most Recent Events

  • 12 Sep 2019 New trial record

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