hrp0097t8 | Section | ESPE2023
M Pimenta Jeanne
, Cohen Shelda
, Mukherjee Swati
, Fettes Fiona
, Jayaram Kala
, Lausch Ekkehart
, Mallya Usha G.
, Min Yang
, Caroline Huber
, Cala Mary Lynn
, Ali Greatsinger
, Jeremy Pomeroy
, Haqq Andrea M.
Introduction: Achondroplasia is caused by a pathogenic mutation in the FGFR3 gene, leading to impaired endochondral bone growth and multiple medical complications. Vosoritide, a modified recombinant human C-type natriuretic peptide (rhCNP), was approved by the European Medicines Agency (EMA) in August 2021 for treating genetically confirmed achondroplasia in patients aged ≥2 years until closure of epiphyses. Acorn is the first treatment-based registry for a...