300 million people living with a rare disease worldwide An open-access article, written by Orphanet (coordinated by INSERM, the French National Institute of Health and Medical Research), EURORDISRare Diseases Europe and Orphanet Ireland (based at the National Rare Diseases Office, Mater Misericordiae University Hospital, Dublin, Ireland), confirms the number of…..
Convergence of digital technology and healthcare – digital health – has become a key driver of change for healthcare systems globally, with the mass adoption of digital technologies set to revolutionise healthcare and patient wellbeing. Payors and healthcare and life science companies must learn from the mistakes made in other…..
FEATURE STORY ● Bluebird bio and Novo Nordisk enter into research agreement to develop in vivo genome editing candidates CLINICAL TRIALS - DATAS ● Risdiplam Spinal Muscular Atrophy data demonstrating continued benefit ● ProQR announces positive top-line results from the phase 1/2 study of Sepofarsen in LCA10 patients ● Ionis'…..
Antibiotic development increased, but still insufficient While the pipeline of new antibiotics has improved over the past six years, momentum in the development of new infection-fighting agents remains inadequate and could take a significant downturn without new incentives, a report released in Clinical Infectious Diseases shows. This report, an update of progress…..
New version of SOPHiA Whole Exome Solution to accelerate rare disease characterization SOPHiA GENETICS, a leader in Data-Driven Medicine, has announced at the American Society of Human Genetics Annual Meeting (ASHG) the release of the new version of its SOPHiA Whole Exome Solution. This advanced genomic application is reducing the…..
$50 million in first close of new advanced therapies fund 4BIO Capital, an international venture capital firm focused solely on the advanced therapies sector, has completed the first close of its second venture fund, 4BIO Ventures II. Over $50 million of capital has been committed from family offices and institutional…..
FEATURE STORY ● The landscape of early clinical gene therapies outside oncology BASIC SCIENCE ● Enhanced CAR T cell expansion and prolonged persistence in pediatric patients with ALL CLINICAL TRIALS/DATA ● ATL1102 for DMD commencement of European regulatory interactions ● UniQure stays a step ahead in hemophilia B FINANCIAL DATA ● Passage Bio Closes $110…..
The 2018 Nobel Prize in Physiology or Medicine has been jointly awarded to James P. Allison and Tasuku Honjo for their discovery of cancer therapy by inhibition of negative immune regulation. During the 1990s, in his laboratory at the University of California, Berkeley, James P. Allison studied the T-cell protein CTLA-4.…..
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