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An Open-Label, Prospective, Interventional Study to Determine the Optimal Treatment of Classical Homocystinuria (HCU) in Infants Identified through Newborn Screening in Qatar

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Trial Profile

An Open-Label, Prospective, Interventional Study to Determine the Optimal Treatment of Classical Homocystinuria (HCU) in Infants Identified through Newborn Screening in Qatar

Status: Planning
Phase of Trial: Phase II

Latest Information Update: 09 Nov 2025

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At a glance

  • Drugs Pegtibatinase (Primary)
  • Indications Homocystinuria
  • Focus Therapeutic Use

Most Recent Events

  • 09 Nov 2025 New trial record

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