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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...
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...
  Collaboration supports manufacture of the OPTF01 product to Drive Next-Generation Chimeric Antigen Receptor T Cell (CAR-T) Development INDIANAPOLIS and EHIME, Japan — Genezen, a leading viral vector Contract Development and Manufacturing Organization (CDMO), and Optieum Biotechnologies, Inc. (Optieum), a preclinical stage company dedicated to the discovery and development of innovative CAR-T cell therapies,...
Lille, France, Cambridge, Massachusetts and Zurich, Switzerland – GENFIT (Euronext: GNFT), a biopharmaceutical company dedicated to improving the lives of patients with rare and life-threatening liver diseases, today reports encouraging preliminary Phase 1b data from its CCA clinical trial evaluating GNS561 in combination. Clinical trial context and objective CCA is a rare...