{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,25]],"date-time":"2026-10-25T00:19:00Z","timestamp":1792887540676,"version":"4.5.0"},"reference-count":21,"publisher":"The Endocrine Society","issue":"1","license":[{"start":{"date-parts":[[2022,9,8]],"date-time":"2022-09-08T00:00:00Z","timestamp":1662595200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100013220","name":"Ultragenyx Pharmaceutical Inc","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013220","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2022,12,17]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:sec>\n                  <jats:title>Context<\/jats:title>\n                  <jats:p>Burosumab was developed as a treatment option for patients with the rare, lifelong, chronically debilitating, genetic bone disease X-linked hypophosphatemia (XLH).<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Objective<\/jats:title>\n                  <jats:p>Collect additional information on the safety, immunogenicity, and clinical response to long-term administration of burosumab.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>UX023-CL203 (NCT02312687) was a Phase 2b, open-label, single-arm, long-term extension study of adult subjects with XLH who participated in KRN23-INT-001 or KRN23-INT-002 studies. The long-term UX023-CL203 study (January 5, 2015 through November 30, 2018) provided data up to 184 weeks. Participants in UX023-CL203 received burosumab based on the last dose in the prior KRN23-INT-001 or KRN23-INT-002 studies (0.3, 0.6, or 1.0\u2005mg\/kg given by subcutaneous injection every 4 weeks). At Week 12, burosumab could be titrated upward\/downward to achieve fasting serum phosphate levels within the normal range. Primary objectives included long-term safety, the proportion of subjects achieving fasting serum phosphate in the normal range, changes in bone turnover markers, patient-reported outcomes for pain and stiffness, and measures of mobility.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>Fasting serum phosphate levels at the midpoint of the dosing interval (2 weeks postdose, the time of peak effect) were within the normal range in 85% to 100% of subjects. Measures of phosphate metabolism and bone biomarkers generally improved with burosumab therapy, approaching or reaching their respective normal ranges by study end. Improvements in patient-reported outcomes and mobility were sustained throughout the observation period. No new safety findings emerged with longer-term burosumab treatment.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusion<\/jats:title>\n                  <jats:p>These data support the conclusion that burosumab therapy may be a safe and effective long-term treatment option for adult patients with XLH.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1210\/clinem\/dgac518","type":"journal-article","created":{"date-parts":[[2022,9,8]],"date-time":"2022-09-08T06:28:52Z","timestamp":1662618532000},"page":"155-165","source":"Crossref","is-referenced-by-count":33,"title":["Long-term Burosumab Administration Is Safe and Effective in Adults With X-linked Hypophosphatemia"],"prefix":"10.1210","volume":"108","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2868-2288","authenticated-orcid":false,"given":"Thomas J","family":"Weber","sequence":"first","affiliation":[{"name":"Duke University , Durham, NC 27708 , 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USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joel","family":"Hetzer","sequence":"additional","affiliation":[{"name":"Ultragenyx Pharmaceutical Inc. , Novato, CA 94949 , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"suffix":"II","given":"J Lawrence","family":"Merritt","sequence":"additional","affiliation":[{"name":"Ultragenyx Pharmaceutical Inc. , Novato, CA 94949 , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karl","family":"Insogna","sequence":"additional","affiliation":[{"name":"Yale University School of Medicine , New Haven, CT 06520 , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"80","published-online":{"date-parts":[[2022,9,8]]},"reference":[{"issue":"7","key":"2022121716463845700_dgac518-B1","doi-asserted-by":"crossref","first-page":"1381","DOI":"10.1002\/jbmr.340","article-title":"A clinician's guide to X-linked 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