These indigenous products will lead to drug prices for rare diseases being slashed up to 100 times of their current market value CHENNAI, India – Providing relief to patients with rare diseases across India, the Union Health Ministry has made available generic drugs to support the care and treatment of...
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PARIS, France – Genethon, a worldwide pioneer and leader in research and development in gene therapy for rare genetic diseases, released positive long-term efficacy data revealing three Duchene muscular dystrophy patients showed significant motor function gain as compared to untreated patients, and experienced significant and sustained reductions in creatine phosphokinase...
PARIS, France — Genethon, a non-profit research organization focused on developing gene therapies for rare diseases, today marks International Rare Disease Day by highlighting its efforts to bring gene therapies to patients suffering from rare diseases such as limb girdle muscular dystrophies, Crigler Najjar syndrome and Duchenne muscular dystrophy. Globally...
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...
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,...