Study of AMT-110 in patients with Sanfilippo B syndrome

Trial Profile

Study of AMT-110 in patients with Sanfilippo B syndrome

Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 04 Apr 2016

At a glance

  • Drugs AMT 110 (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Adverse reactions; Therapeutic Use
  • Most Recent Events

    • 04 Apr 2016 According to a media release, uniQure expects to present 30-month follow-up data from four patients in early 2017.
    • 19 Sep 2015 Results presented at the European Society of Gene and Cell Therapy (ESGCT) and Finnish Society of Gene Therapy (FSGT) Collaborative Congress, as per Uniqure media release.
    • 19 Sep 2015 Positive top-line results published in Uniqure media release.
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