Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension

OBJECTIVES: To report the impact of burosumab on patient-reported outcomes (PROs) and ambulatory function in adults with X-linked hypophosphataemia (XLH) through 96 weeks. METHODS: Adults diagnosed with XLH were randomised 1:1 in a double-blinded trial to receive subcutaneous burosumab 1 mg/kg or pl...

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Detalles Bibliográficos
Autores principales: Briot, Karine, Portale, Anthony A, Brandi, Maria Luisa, Carpenter, Thomas O, Cheong, Hae Ii, Cohen-Solal, Martine, Crowley, Rachel K, Eastell, Richard, Imanishi, Yasuo, Ing, Steven, Insogna, Karl, Ito, Nobuaki, Jan de Beur, Suzanne, Javaid, Muhammad K, Kamenicky, Peter, Keen, Richard, Kubota, Takuo, Lachmann, Robin H, Perwad, Farzana, Pitukcheewanont, Pisit, Ralston, Stuart H, Takeuchi, Yasuhiro, Tanaka, Hiroyuki, Weber, Thomas J, Yoo, Han-Wook, Nixon, Annabel, Nixon, Mark, Sun, Wei, Williams, Angela, Imel, Erik A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2021
Materias:
Treatments
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8458321/
https://www.ncbi.nlm.nih.gov/pubmed/34548383
http://dx.doi.org/10.1136/rmdopen-2021-001714

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