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Potential Registration enabling study of VOXZOGO (Vosoritide) in idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency

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Trial Profile

Potential Registration enabling study of VOXZOGO (Vosoritide) in idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency

Status: Planning
Phase of Trial: Phase III

Latest Information Update: 22 Nov 2025

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At a glance

  • Drugs Vosoritide (Primary)
  • Indications Noonan syndrome; Short stature; Turner's syndrome
  • Focus Registrational; Therapeutic Use

Most Recent Events

  • 22 Nov 2025 New trial record
  • 27 Oct 2025 According to a BioMarin media release, 4 new VOXZOGO indications are under development as part of BioMarin's CANOPY clinical program, which include idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency, with potential registration-enabling studies in 2027.

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