PARIS, France — Genethon, a worldwide pioneer and leader in research and development in gene therapy for rare genetic diseases, has received approvals from regulatory authorities, MHRA and EMA*, to begin pivotal Phase 3 clinical trials in France and the UK of its gene therapy, GNT0004, for Duchenne muscular dystrophy...
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BUFFALO, NY – A new research perspective was published in Oncotarget, entitled, “Genetic alterations in thyroid cancer mediating both resistance to BRAF inhibition and anaplastic transformation.” In this new paper, researchers Mark Lee and Luc GT Morris from New York Presbyterian Hospital and Memorial Sloan Kettering Cancer Center discuss thyroid...
BUFFALO, NY – A new research paper was published in Oncotarget’s Volume 15 entitled “Genetic and therapeutic landscapes in cohort of pancreatic adenocarcinomas: next-generation sequencing and machine learning for full tumor exome analysis.” About 7% of all cancer deaths are caused by pancreatic cancer (PCa). PCa is known for its...
Toronto, Ontario – A Canadian-led study has identified genes which may be partially responsible for the development of cerebral palsy. Cerebral palsy (CP), a condition that affects the development of motor skills in children, is the most common childhood-onset physical disability. CP can have different causes, such as infections, injuries,...
Scientists have found genetic variations that appear to increase the risk of developing primary biliary cirrhosis, a baffling disease that can destroy the liver. Primary biliary cirrhosis is an autoimmune disorder that blocks the bile ducts in the liver. It is a condition that mostly affects women, striking about one...
A team led by Nagoya University Graduate School of Medicine in Japan has investigated screen time in autistic (ASD) and ADHD children. Screen time refers to the amount of time a person spends on a device with a screen, such as a smartphone, computer, television, or video game system. The...
As per the Organization of Rare diseases in India (ORDI), 1 in 20 Indians is affected by a rare disorder. More than 7,000 rare diseases are known and reported worldwide; from these approximately 80 per cent are known to have a genetic predisposition. Some of these “common” rare diseases we’ve...
A collaboration research carried out by the teams of Jordi Surrallés, Universitat Autònoma de Barcelona (UAB); Juan Carlos Izpisúa-Belmonte and Ángel Raya, Centre for Regenerative Medicine of Barcelona (CMRB); and Juan Antonio Bueren, Centre for Energetic, Environmental and Technological Research (CIEMAT), has resulted in the generation of blood cells from...
BEIJING – Genetron Holdings Limited (“Genetron Health” or the “Company”, NASDAQ:GTH), a leading precision oncology platform company in China that specializes in molecular profiling tests, early cancer screening products and companion diagnostics development, presented new data and results for HCCscreen™, a blood-based early screening test for hepatocellular carcinoma (“HCC”). This...
SARASOTA, Fla. and NOVATO, Calif. – GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced that they have received clearance from Health Canada to begin enrolling the Phase 1/2 study of GTX-102 in...