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

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