News BioMarin halts Voxzogo trials due to safety signal BioMarin has suspended dosing in phase 2 trials of its achondroplasia therapy Voxzogo aimed at broadening the drug's label.
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 BioMarin ends year with $4.8bn play for Amicus BioMarin takes a leap towards its $4bn revenue target with a $4.8bn deal to acquire Amicus, which has two rare disease therapies on the market.
News NHS cleared to use AZ's Fasenra for rare autoimmune disease NICE backs NHS use of an AstraZeneca drug for one rare disease, but says it can't recommend a BioMarin therapy for another.
News BioMarin starts rebuilding after its downsizing spree BioMarin has made its first M&A play under James Sabry, buying Inozyme to flesh out its enzyme replacement therapies (ERTs) for rare diseases.
News Spruce Bio sows the seeds of its new future Spruce has bounced back from the failure of its lead drug last year, thanks to a licensing deal with BioMarin for a late-stage Sanfilippo drug.
News OpenBind unveils its first AI model for drug discovery A research consortium that aims to make the UK a leader in AI-driven drug discovery, OpenBind, has hit its first major milestone.
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