Trial Profile

Study of AMT-110 in patients with Sanfilippo B syndrome

Status: Active, no longer recruiting

Phase of Trial: Phase I/II

Latest Information Update: 06 Apr 2016

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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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