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phase 3 trial of vosoritide in children with hypochondroplasia
Original title: “A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia.”
phase 3 randomized double blind placebo controlled multicenter study to evaluate the efficacy and safety of vosoritide in children with hypochondroplasia canopy hch 3 study design
Original title: “A phase 3, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of vosoritide in children with hypochondroplasia: CANOPY HCH-3 study design.”
diagnosis of hypochondroplasia international delphi consensus recommendations
Original title: “Diagnosis of hypochondroplasia: international Delphi consensus recommendations.”
natural history of hypochondroplasia a retrospective matched cohort study using the clinical practice research datalink cprd aurum database
Original title: “Natural history of hypochondroplasia: A retrospective, matched-cohort study using the clinical practice research Datalink (CPRD) aurum database.”
After being hit by safety issues and subpar results in another trial, BioMarin’s Phase 3 test of Voxzogo for a rare skeletal disorder called hypochondroplasia showed efficacy “solidly above” what the drug has shown for achondroplasia, which causes dwarfism.
Original title: “BioMarin bounces back as Voxzogo performs ‘bigger than expected’ in new indication”
pathways to facilitate early recognition and diagnosis of hypochondroplasia
Original title: “Pathways to Facilitate Early Recognition and Diagnosis of Hypochondroplasia.”