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’s Voxzogo shows promise in hypochondroplasia BioMarin’s Voxzogo has been shown to increase growth rates in children with hypochondroplasia, a genetic cause of very short stature, in a phase 2 trial.
News Chiesi signs $1.9bn deal to buy KalVista and its HAE drug Chiesi has boosted its rare disease business with an agreement to buy KalVista and Ekterly, its oral drug for treating attacks in rare disease HAE.
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