News BridgeBio gains on phase 3 dwarfism data Shares in BridgeBio rose after it revealed data from a trial of infigratinib in achondroplasia, raising the prospect of the first oral treatment.
News BridgeBio two for two as rare disease drug aces ph3 trial BridgeBio's week just went from great to superb, as it reports a second positive topline result in a pivotal trial of a late-stage pipeline drug.
News Alnylam joins three-way contest in ATTR cardiomyopathy Alnylam has claimed FDA approval for Amvuttra in ATTR cardiomyopathy, allowing it to compete with rival therapies from Pfizer and BridgeBio.
News BridgeBio gets EU nod for ATTR cardiomyopathy drug BridgeBio and Bayer are poised to challenge Pfizer's Vyndaqel in the EU after acoramidis gets a green light for ATTR cardiomyopathy.
News BridgeBio poised to challenge Pfizer after Attruby approval BridgeBio grabs FDA approval for its ATTR cardiomyopathy drug Attruby, setting up a challenge to market leader Pfizer
News Alnylam hails vutrisiran data, but investors seem sceptical Alnylam Pharma has been riding high since revealing top-line results from its HELIOS-B trial of vutrisiran in transthyretin-mediated amyloidosis (ATTR) – but a look at the full data seems t
News NHS "isn't delivering equitable care for rare diseases" A Genetic Alliance UK report claims the NHS is failing to meet the needs of many of the 3.5 million people in the country who live with rare diseases.
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