Research Project

Project Title:	Compassionate use of Crysvita (burosumab), a recombinant human monoclonal antibody (IgG1) inhibitor of fibroblast growth factor 23 activity, for the treatment of X-linked hypophosphatemia in adult patients with radiographic evidence of bone disease
Project Type:	Observational Study, Enrolment terminated
Disease group(s):	Tubulopathies
Project Summary:	The main purpose of the compassionate use is to facilitate access to Crysvita® (burosumab) for Adult patients with X-Linked Hypophosphataemia (XLH) who lack therapeutic alternatives, are in immediate need for treatment and in which there is a reasonable expectation that the provision of access will have a positive benefit/risk for the patient.
Lead principal investigator(s):	Marica Giliberti, Bari
Co-investigator(s):	S Russo, Bari
Project Period:	01/2020 - 12/2023
Sponsors:	Industry