Real-world data of Brazilian adults with X-linked hypophosphatemia (XLH) treated with burosumab and comparison with other worldwide cohorts.

Maria Helena Vaisbich Antônio César Paulillo de Cillo Bárbara Campolina C Silva Catarina Brasil DÁlva Érico Higino de CarvalhoJuliana M C M de AlmeidaLarissa L M MarquesMarcia Gonçalves Ribeiro Mauro Borghi M da Silva Paula Frassinetti Vasconcelos de Medeiros Pedro Henrique Mendes

Published in: Molecular genetics & genomic medicine (2024)

This study confirms the efficacy and safety of burosumab on XLH adult patients observed in clinical trials. Additionally, we observed a decrease in iPTH levels in patients with moderate to severe HPT at the baseline.

Keyphrases

clinical trial
early onset
randomized controlled trial
machine learning
data analysis
newly diagnosed
artificial intelligence
study protocol