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Los Angeles, Calif. – Investigators at the UCLA Health Jonsson Comprehensive Cancer Center have uncovered new details about the role of a protein called interferon regulatory factor (IRF1) in cancer progression and treatment response, offering new insight that can potentially help improve the effectiveness of cancer immunotherapy. The study, published in Cell Reports, reveals...
A team led by a University of Florida Health scientist last month landed what it believes is the first National Institutes of Health grant ever awarded specifically to study a rare neurological disease that affects the lives of children and their families.
GAINESVILLE — A dog born with a deadly disease that prevents the body from using stored sugar has survived 20 months and is still healthy after receiving gene therapy at the University of Florida — putting scientists a step closer to finding a cure for the disorder in children. Called...
PARIS – GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that its gene therapy LUMEVOQ® has been granted Promising Innovative Medicine (PIM) designation by the UK’s Medicines and...
The son of Vitaliy Svichynskyi and Maria Svichynska was nearly two months old when he suddenly started to lose the ability to move. The family soon learned their baby boy Dmytro was born with Spinal Muscular Atrophy (SMA) Type 1, the most severe type of the rare genetic disorder found in...
Ultimovacs has announced that the U.S. Food and Drug Administration has granted orphan drug designation (ODD) for the company’s universal cancer vaccine UV1 for the treatment of stage IIB – IV melanoma. UV1, as an add-on therapy to checkpoint inhibitors ipilimumab and nivolumab, is currently being studied as a first-line...
Ultomiris (ravulizumab) has been recommended for marketing authorisation in the European Union (EU) for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive (Ab+). If authorised, Ultomiris would be the first and only approved long-acting C5 complement inhibitor for the treatment of...