Data from an investigator-led study showed treatment with VOXZOGO resulted in sustained improvements in growth over three years in children with hypochondroplasia ....
New Phase 1 results also presented for investigational BMN333 support potential weekly dosing in children with achondroplasia ... New BMN 333 Phase 1 DataSupport Advancement Into Late-Stage Development.
BMRN) today announced results from the pivotal Phase 3 ENERGY 3 trial evaluating BMN 401 in children aged 1-12 with ENPP1 deficiency, a rare, serious and progressive genetic condition ... About BMN 401 and ENERGY 3.