rare - International Biopharmaceutical Industry

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FDA places stricter limits on J&J’s COVID-19 vaccine after review of rare side effect

Dive Brief: The Food and Drug Administration has determined that the risk of a rare, but serious, clotting syndrome tied to Johnson & Johnson’s COVID-19 vaccin...

9 May 2022 news COVID-19 | FDA

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As biotech retreats, gene therapy companies retrench and redraw plans

Bluebird bio has spent the better part of two decades developing gene therapies for rare diseases. This summer, after years of clinical trials, setbacks and delays, t...

25 March 2022 news Biotech | companies

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Star Therapeutics emerges with plans to treat rare diseases through a solar system of drug companies

Few things are as human as gazing into the night sky. And yet, after thousands of years, we know only a little about what we're looking at and how it all works. Spac...

18 February 2022 news diseases | rare

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In England, Orchard secures rare agreement on gene therapy coverage

Dive Brief: The National Health Service in England and Wales will cover treatment with an exceptionally high-priced gene therapy for a rare and fatal genetic disea...

6 February 2022 news coverage | gene

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Novartis hits another setback in plan to repurpose rare disease drug in lung cancer

Novartis' testing of canakinumab, which works by curbing inflammatory signaling in cells, for the seemingly unrelated use in lung cancer stems from an intriguing 2011...

29 October 2021 news cancer | cells

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