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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 A new Fabry disease treatment The agreement made in the NICE’s Final Draft Guidance means there is now a “new treatment option for people living with Fabry...
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...
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...
Bethesda, MD – Three different HIV antibodies each independently protected monkeys from acquiring simian-HIV (SHIV) in a placebo-controlled proof-of-concept study intended to inform development of a preventive HIV vaccine for people. The antibodies—a human broadly neutralizing antibody and two antibodies isolated from previously vaccinated monkeys—target the fusion peptide, a site...