A phase I/II, open-label, study of intracerebral administration of adeno-associated viral vectors carrying human NaGlu cDNA for the treatment of Sanfilippo type B syndrome

Trial Profile

A phase I/II, open-label, study of intracerebral administration of adeno-associated viral vectors carrying human NaGlu cDNA for the treatment of Sanfilippo type B syndrome

Completed
Phase of Trial: Phase I/II

Latest Information Update: 05 Oct 2017

At a glance

  • Drugs AMT 110 (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus First in man; Therapeutic Use
  • Most Recent Events

    • 23 Sep 2017 New trial record
    • 01 Sep 2017 Results published in the Lancet Neurology
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