The objectives of this observational study are to characterize XLH disease presentation and progression and to assess long-term effectiveness and safety of burosumab.
Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.
Ultragenyx Pharmaceutical (RARE) is the sponsoring company for this trial. BiotechSign currently grades this company D (49/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.