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Positive Topline Results Announced from Phase I/II Trial in Sanfilippo B Syndrome Patients Using uniQure’s Novel AAV5-Based Gene Therapy

September 21, 2015

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uniQure N.V. (Nasdaq: QURE), a leader in human gene therapy, today announced the topline results of one-year follow-up data from a Phase I/II clinical trial conducted by Institut Pasteur (Biotherapies for Neurodegenerative Diseases Unit, Institut Pasteur/INSERM) in partnership with the French Muscular Dystrophy Association and Vaincre les Maladies Lysosomales (collectively “the consortium”) in four Sanfilippo Bsyndrome (MPSIIIB) patients treated with a novel gene therapy, AMT-110.