News Ascendis scores FDA approval for weekly dwarfism drug Children with achondroplasia will soon have the option of a once-weekly - rather than daily - injection, courtesy of Ascendis' newly approved Yuviwel.
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 Pfizer acquires Therachon and its dwarfism drug Pfizer has announced it will pay $340 million upfront to snap up rare disease biotech Therachon, which is developing treatments for a form
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.
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