NICE’s draft guidance published on 8 March 2021 recommends £1.79 million treatment Zolgensma (also called onasemnogene abeparvovec and made by Novartis Gene Therapies) for babies aged up to 12 months with type 1 SMA, This is one of the severest forms of the inherited condition and the life-expectancy of people with...
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The British National Institute for Health and Care Excellence (NICE) has recommended Chiesi’s Elfabrio® (pegunigalsidase alfa) for Fabry disease (alpha-galactosidase deficiency) in adults. A new Fabry disease treatment The agreement made in the NICE’s Final Draft Guidance means there is now a “new treatment option for people living with Fabry...
DETROIT, Michigan – A Wayne State University College of Engineering professor has received a $2.65 million award from the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health to develop a novel filtration platform to improve an advanced drug delivery device to optimize diabetes...
North Bethesda, Maryland – A trial of a new drug regimen to treat tuberculous meningitis (TBM) has started enrolling adults and adolescents in several countries where tuberculosis (TB) is prevalent. The Improved Management with Antimicrobial Agents Isoniazid Rifampicin Linezolid for TBM (IMAGINE-TBM) trial will compare a six-month regimen of four...
WHO: The Gabriella Miller Kids First Pediatric Research Program (Kids First), an initiative of the National Institutes of Health (NIH) WHAT: Kids First announces the release of three comprehensive new pediatric research datasets exploring childhood cancers and congenital disorders. New publicly available datasets include: CHILDHOOD CANCERS Gabriella Miller Kids...
Philadelphia, PA. WHO: The Gabriella Miller Kids First Pediatric Research Program (Kids First), an initiative of the National Institutes of Health (NIH) WHAT: Kids First announces the release of nine robust new pediatric research datasets spanning childhood cancers, congenital disorders, and cross-condition data. New publicly available datasets include: PEDIATRIC CROSS-CONDITION...
Scientists at the National Institutes of Health have gained a major insight into how the rogue protein responsible for mad cow disease and related neurological illnesses destroys healthy brain tissue. “This advance sets the stage for future efforts to develop potential treatments for prion diseases or perhaps to prevent them...
Bethesda, Maryland – In a proof-of-concept study, researchers at the National Institutes of Health (NIH) have developed an artificial intelligence (AI) tool that uses data from individual cells inside tumors to predict whether a person’s cancer will respond to a specific drug. Researchers at the National Cancer Institute (NCI), part...
Bethesda, Maryland – Researchers at the National Institutes of Health (NIH) and their collaborators have discovered a new way in which RAS genes, which are commonly mutated in cancer, may drive tumor growth beyond their well-known role in signaling at the cell surface. Mutant RAS, they found, helps to kick...
High-risk research into neglected diseases, which can often fail at the first hurdle, may be made safer as a result of a new programme. The Therapeutics for Rare and Neglected Diseases (TRND) Programme will support preclinical research in which candidate drugs are tested in cells or animals — one of...