A first in man clinical study of autologus ex-vivo lentiviral gene therapy for the treatment of mucopolysaccharidosis type IIIA

Trial Profile

A first in man clinical study of autologus ex-vivo lentiviral gene therapy for the treatment of mucopolysaccharidosis type IIIA

Planning
Phase of Trial: Phase I

Latest Information Update: 24 Oct 2016

At a glance

  • Drugs SGSH gene therapy-Orchard Therapeutics (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Adverse reactions; First in man
  • Most Recent Events

    • 24 Oct 2016 New trial record
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