LEXINGTON, Mass. and AMSTERDAM, Netherlands— uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the company reached agreement with the U.S. Food and Drug Administration (FDA) on key elements of an Accelerated Approval pathway for AMT-130. “We are...
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LEXINGTON, Mass. and AMSTERDAM — uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated interim data including up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for...
Nijmegen, The Netherlands – TB is a major threat to public health, being among the leading causes of death worldwide. In 2021, the disease claimed the lives of 1.6 million people, making it the second leading infectious killer after COVID-19.[1] Drug-resistant TB and long treatment regimens have increased the urgency...
Birmingham, England – Societal acceptance of autism varies considerably across different countries, with lowest levels of acceptance found in Japan and Belgium, new research shows. A survey of 306 autistic individuals from eight countries revealed that around three quarters of respondents do not feel accepted, or only sometimes feel accepted,...
OKLAHOMA CITY, Okla. – In a study published Wednesday in the Proceedings of the National Academy of Sciences (PNAS), University of Oklahoma researchers detail their discoveries about why the brain tumor glioblastoma is so aggressive. Their findings center on ZIP4, a protein that transports zinc throughout the body and sets...
Southampton, UK – Scientists have taken a major step towards developing a blood test that could identify millions of people who spread tuberculosis unknowingly. A breakthrough study has discovered a group of biological markers that are found in high levels among infectious patients. The researchers hope the findings will pave...
Toronto, Canada – Researchers at the University of Toronto and Sinai Health have created a new platform to identify proteins that can be co-opted to control the stability of other proteins — a new but largely unrealized approach to the treatment of disease. The researchers developed a method to interrogate the...
Tokyo, Japan – The onset of psychosis can be predicted before it occurs, using a machine-learning tool which can classify MRI brain scans into those who are healthy and those at risk of a psychotic episode. An international consortium including researchers from the University of Tokyo, used the classifier to...
BUFFALO, N.Y. — A viral gene therapy developed by University at Buffalo researchers has reversed some brain abnormalities in infant mice with FOXG1 syndrome, a significant step toward one day treating children with this severe neurodevelopmental disorder. This mediated delivery of the FOXG1 gene via adeno-associated virus 9 (AAV9) is...
BUFFALO, N.Y. — More than a decade ago, physicians around the world began reporting cases due to a new hypervirulent strain of Klebsiella pneumoniae, which could infect and severely sicken otherwise healthy people. Thomas A. Russo, MD, at the University at Buffalo and the VA Western New York Healthcare System...