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~ Treatment generally well-tolerated with no significant safety issues related to AMT-130 in treated patients through one year of follow-up ~ ~ A mean reduction of 53.8% of mutant HTT (mHTT) observed...

~ Treatment was well tolerated with no significant safety issues related to AMT-130 in first two treated patients through one year of follow-up ~ ~ Neurofilament Light Chain (NfL) rose as expected...

LEXINGTON, Mass. and AMSTERDAM, The Netherlands, June 16, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with...

LEXINGTON, Mass. and AMSTERDAM, The Netherlands, May 27, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with...

~ No Significant Safety Concerns Observed ~ ~ Independent Data Safety Monitoring Board Recommends Proceeding with Study Enrollment ~ ~ Full Enrollment of First Cohort Expected Mid-2021 ~ LEXINGTON,...

LEXINGTON, Mass. and AMSTERDAM, June 19, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical...

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 15, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with...

~ Mutant Huntingtin Protein Reduced by a Median of 68% in the Deep Structures of the Brain and a Median of 47% in the Frontal Cortex at 6 Months in a Large Diseased Animal Model ~   LEXINGTON, Mass....

NOVATO, Calif., March 21, 2016 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. (Nasdaq:RPTP) today announced that Health Canada has accepted for review its New Drug Submission (NDS) for PROCYSBI®...

NOVATO, Calif., March 01, 2016 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. (Nasdaq:RPTP) today announced that it will participate in the following investor conference: Julie Anne Smith,...