EVRY, France — Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular dystrophy in patients treated at therapeutic doses in the first phase of an international multicenter clinical trial...
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GNT0004 product confirms its clinical efficacy with stabilization of motor functions in patients treated at the effective dose for up to 2 years. Sustained significant reduction in levels of creatine phosphokinase (CPK), a biomarker of muscle damage, with an average decrease of over 75% at 18 months in the 3 patients treated...
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...
PARIS, France – Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today its research and clinical trial data will be featured in eight oral presentations and 13 posters at the American Society of Gene & Cell Therapy Annual Meeting May 11-15...
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...