{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,7]],"date-time":"2026-04-07T08:32:45Z","timestamp":1775550765565,"version":"3.50.1"},"reference-count":79,"publisher":"Georg Thieme Verlag KG","issue":"10","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Horm Metab Res"],"published-print":{"date-parts":[[2022,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>The last decade has been revolutionary regarding the management of rare bone diseases caused by impaired calcium and phosphate metabolism. Elucidation of the underlying genetic basis and pathophysiologic alterations has been the determinant factor for the development of new, disease-specific treatment agents. The phosphaturic hormone Fibroblast Growth Factor 23 (FGF23) possesses a critical role in the pathogenesis of various hypophosphatemic disorders. Among them, the genetic disorder of X-linked hypophosphatemia and the acquired syndrome of tumor-induced osteomalacia, although very rare, have attracted the scientific community\u2019s attention towards designing an FGF23-inhibitor as a potential specific therapy. The monoclonal antibody burosumab was approved for the treatment of children and adult patients with X-linked hypophosphatemia and recently for tumor-induced osteomalacia patients, demonstrating benefits regarding their symptoms, biochemical profile and bone mineralization status. Asfotase alfa is a hydroxyapatite-targeted recombinant alkaline phosphatase, an enzymatic replacement therapy, substituting the defective activity of tissue non-specific alkaline phosphatase, in patients suffering from hypophosphatasia. Promising data regarding its favorable effect on survival rate, bone quality, fracture healing, muscle strength, mobility, respiratory function, and general quality of life have led to the approval of the drug for the treatment of childhood-onset hypophosphatasia. Given the high costs of treatment for both agents and their limited clinical use until now, more data are needed to define patients\u2019 characteristics that make them ideal candidates for therapy. Long-term safety issues also need to be clarified.<\/jats:p>","DOI":"10.1055\/a-1917-0519","type":"journal-article","created":{"date-parts":[[2022,9,1]],"date-time":"2022-09-01T23:51:26Z","timestamp":1662076286000},"page":"645-657","source":"Crossref","is-referenced-by-count":3,"title":["Novel Therapeutic Agents for Rare Diseases of Calcium and Phosphate Metabolism"],"prefix":"10.1055","volume":"54","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9803-7789","authenticated-orcid":false,"given":"Afroditi","family":"Roumpou","sequence":"additional","affiliation":[{"name":"Division of Endocrinology, Metabolism and Diabetes, First Department of Pediatrics, \u201cAghia Sofia\u201d Children\u2019s Hospital, National and Kapodistrian University of Athens School of Medicine, Athens, Greece"},{"name":"Laboratory for Research of the Musculoskeletal System, National and Kapodistrian University of Athens School of Medicine, Athens, Greece"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria P.","family":"Yavropoulou","sequence":"additional","affiliation":[{"name":"Centre of Expertise for Rare Endocrine Diseases, C.E.R.E.D. Disorders of Calcium & Phosphate Metabolism, First Department of Propaedeutic and Internal Medicine, Laiko Hospital, National and Kapodistrian University of Athens School of Medicine, Athens, Greece"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Efstathios","family":"Chronopoulos","sequence":"additional","affiliation":[{"name":"Laboratory for Research of the Musculoskeletal System, National and Kapodistrian University of Athens School of Medicine, Athens, Greece"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eva","family":"Kassi","sequence":"additional","affiliation":[{"name":"Laboratory for Research of the Musculoskeletal System, National and Kapodistrian University of Athens School of Medicine, Athens, Greece"},{"name":"Centre of Expertise for Rare Endocrine Diseases, C.E.R.E.D. 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Addison","year":"2010","journal-title":"J Bone Miner Res"},{"key":"ref7","doi-asserted-by":"crossref","first-page":"688","DOI":"10.1002\/jbmr.1766","article-title":"Proteolytic processing of osteopontin by PHEX and accumulation of osteopontin fragments in Hyp mouse bone, the murine model of X-linked hypophosphatemia","volume":"28","author":"NM T Barros","year":"2013","journal-title":"J Bone Miner Res"},{"key":"ref8","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1007\/s10354-020-00810-w","article-title":"New therapeutic options for bone diseases","volume":"171","author":"R Kocijan","year":"2021","journal-title":"Wien Med Wochenschr"},{"key":"ref9","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.jocd.2019.09.006","article-title":"Proceedings of the 2019 Santa Fe bone symposium: new concepts in the care of osteoporosis and rare bone diseases","volume":"23","author":"E M Lewiecki","year":"2020","journal-title":"J Clin 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Fratzl-Zelman","year":"2020","journal-title":"J Struct Biol"},{"key":"ref14","doi-asserted-by":"crossref","first-page":"338","DOI":"10.3389\/fendo.2020.00338","article-title":"Clinical evidence for the benefits of burosumab therapy for X-linked hypophosphatemia (XLH) and other conditions in adults and children","volume":"11","author":"A Schindeler","year":"2020","journal-title":"Front Endocrinol (Lausanne)"},{"key":"ref15","doi-asserted-by":"crossref","first-page":"469","DOI":"10.1002\/jbmr.3903","article-title":"Nonremission and recurrent tumor-induced osteomalacia: a retrospective study","volume":"35","author":"X Li","year":"2020","journal-title":"J Bone Miner Res"},{"key":"ref16","doi-asserted-by":"crossref","first-page":"10178","DOI":"10.1002\/jbm4.10178","article-title":"Successful management of tumor-induced osteomalacia with radiofrequency ablation: a case series","volume":"3","author":"S K Mishra","year":"2019","journal-title":"JBMR 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Med"},{"key":"ref40","doi-asserted-by":"crossref","first-page":"4303","DOI":"10.3390\/ijms22094303","article-title":"Hypophosphatasia: a unique disorder of bone mineralization","volume":"22","author":"J M Villa-Su\u00e1rez","year":"2021","journal-title":"Int J Mol Sci"},{"key":"ref41","doi-asserted-by":"crossref","first-page":"5676","DOI":"10.3390\/jcm10235676","article-title":"Hypophosphatasia","volume":"10","author":"S Tournis","year":"2021","journal-title":"J Clin Med"},{"key":"ref42","doi-asserted-by":"crossref","first-page":"144855","DOI":"10.1016\/j.gene.2020.144855","article-title":"Alkaline phosphatase: structure, expression and its function in bone mineralization","volume":"754","author":"S Vimalraj","year":"2020","journal-title":"Gene"},{"key":"ref43","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1272\/jnms.77.4","article-title":"The mechanism of mineralization and the role of alkaline phosphatase in health and disease","volume":"77","author":"H Orimo","year":"2010","journal-title":"J Nippon Med School"},{"key":"ref44","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1038\/ng0995-45","article-title":"Mice lacking tissue non-specific alkaline phosphatase die from seizures due to defective metabolism of vitamin B-6","volume":"11","author":"K G Waymire","year":"1995","journal-title":"Nat Genet"},{"key":"ref45","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1007\/s11926-018-0778-5","article-title":"Hypophosphatasia: from diagnosis to treatment","volume":"20","author":"S Simon","year":"2018","journal-title":"Curr Rheumatol Rep"},{"key":"ref46","first-page":"164","article-title":"Hypophosphatasia \u2013 pathophysiology and treatment","volume":"8","author":"J L Mill\u00e1n","year":"2012","journal-title":"Actual Osteol"},{"key":"ref47","doi-asserted-by":"crossref","first-page":"667","DOI":"10.1002\/jbmr.3075","article-title":"Hypophosphatasia: enzyme replacement therapy brings new opportunities and new 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Metab"},{"key":"ref51","first-page":"266","article-title":"Teriparatide treatment in adult hypophosphatasia in a patient exposed to bisphosphonate: a case report","volume":"6","author":"K B Doshi","year":"2009","journal-title":"Clin Cases Miner Bone Metab"},{"key":"ref52","doi-asserted-by":"crossref","first-page":"250","DOI":"10.1007\/s00223-011-9562-5","article-title":"Failure of teriparatide in treatment of bone complications of adult hypophosphatasia","volume":"90","author":"M Laroche","year":"2012","journal-title":"Calcif Tissue Int"},{"key":"ref53","doi-asserted-by":"crossref","first-page":"1007","DOI":"10.1210\/jc.2009-1965","article-title":"Lack of sustained response to teriparatide in a patient with adult hypophosphatasia","volume":"95","author":"C Gagnon","year":"2010","journal-title":"J Clin Endocrinol Metab"},{"key":"ref54","doi-asserted-by":"publisher","first-page":"1759720X19863997","DOI":"10.1177\/1759720X19863997","article-title":"Update on the management of hypophosphatasia","volume":"11","author":"V Choida","year":"2019","journal-title":"Ther Adv Musculoskelet Dis"},{"key":"ref55","doi-asserted-by":"crossref","first-page":"48","DOI":"10.1177\/1759720X13510479","article-title":"Role of sclerostin in bone and cartilage and its potential as a therapeutic target in bone diseases","volume":"6","author":"E M Lewiecki","year":"2014","journal-title":"Ther Adv Musculoskelet Dis"},{"key":"ref56","doi-asserted-by":"crossref","first-page":"860","DOI":"10.1359\/jbmr.080216","article-title":"Targeted deletion of the sclerostin gene in mice results in increased bone formation and bone strength","volume":"23","author":"X Li","year":"2008","journal-title":"J Bone Miner Res"},{"key":"ref57","first-page":"412","article-title":"Romosozumab in postmenopausal women with low bone mineral density. N Eng","volume":"370","author":"M R McClung","year":"2014","journal-title":"J Med"},{"key":"ref58","doi-asserted-by":"crossref","first-page":"2148","DOI":"10.1172\/JCI83731","article-title":"Efficacy of anti-sclerostin monoclonal antibody BPS804 in adult patients with hypophosphatasia","volume":"127","author":"L Seefried","year":"2017","journal-title":"J Clin Invest"},{"key":"ref59","doi-asserted-by":"crossref","first-page":"2015","DOI":"10.1359\/jbmr.1999.14.12.2015","article-title":"Alkaline phosphatase knock-out mice recapitulate the metabolic and skeletal defects of infantile hypophosphatasia","volume":"14","author":"K N Fedde","year":"1999","journal-title":"J Bone Miner Res"},{"key":"ref60","doi-asserted-by":"crossref","first-page":"777","DOI":"10.1359\/jbmr.071213","article-title":"Enzyme replacement therapy for murine hypophosphatasia","volume":"23","author":"J L Mill\u00e1n","year":"2008","journal-title":"J Bone Miner Res"},{"key":"ref61","doi-asserted-by":"crossref","first-page":"470","DOI":"10.1177\/0022034510393517","article-title":"Enzyme replacement therapy prevents dental defects in a model of hypophosphatasia","volume":"90","author":"M D McKee","year":"2011","journal-title":"J Dent Res"},{"key":"ref62","doi-asserted-by":"crossref","first-page":"904","DOI":"10.1056\/NEJMoa1106173","article-title":"Enzyme-replacement therapy in life-threatening hypophosphatasia","volume":"366","author":"M P Whyte","year":"2012","journal-title":"N Engl J Med"},{"key":"ref63","doi-asserted-by":"crossref","first-page":"93","DOI":"10.1016\/S2213-8587(18)30307-3","article-title":"Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial","volume":"7","author":"M P Whyte","year":"2019","journal-title":"Lancet Diabetes 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