Rare Diseases Clinical Trial
Official title:
12-months of Treatment With Burosumab in Children and Adolescents With X-linked Hypophosphatemia: a Prospective Longitudinal Cohort Study
In this prospective longitudinal cohort study we studied the efficacy and safety of burosumab
in real-clinical practice for <13- and >13-years old children affected with X-linked
hypophosphatemia.
57 children with XLH were switched from conventional treatment to burosumab. After 12 months
we assessed the efficacy and safety of treatment with burosumab on the whole cohort and
separately on the cohort of >13-years old adolescents.
n/a
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