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 Merck rare tumour drug Ezmekly backed for NHS use Merck's drug for tumours associated with rare disease neurofibromatosis type 1, Ezmekly, has been cleared for NHS use in children as young as two.
Digital Sponsored Discover a new era of healthcare panel engagement: Grounded ... A new webinar from Konovo will address some of the critical topics impacting today’s healthcare market research industry.
