{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,2,4]],"date-time":"2025-02-04T09:40:25Z","timestamp":1738662025139,"version":"3.35.0"},"reference-count":15,"publisher":"Springer Science and Business Media LLC","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025,2,4]]},"DOI":"10.7759\/cureus.78473","type":"journal-article","created":{"date-parts":[[2025,2,4]],"date-time":"2025-02-04T09:02:31Z","timestamp":1738659751000},"source":"Crossref","is-referenced-by-count":0,"title":["Long-Term Outcomes of Early Enzyme Replacement Therapy With Asfotase Alfa in Perinatal Benign Hypophosphatasia: Amelioration of Bone Deformities in a Young Child"],"prefix":"10.7759","author":[{"given":"Shuntaro","family":"Terayama","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Masahisa","family":"Kobayashi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tokumasa","family":"Suemitsu","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Osamu","family":"Samura","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kimihiko","family":"Oishi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","reference":[{"key":"ref1","article-title":"Hypophosphatasia","volume":"10","author":"Rockman-Greenberg C","year":"2013","unstructured":"Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev. 2013, 10:380-8.","journal-title":"Pediatr Endocrinol Rev"},{"key":"ref2","doi-asserted-by":"publisher","DOI":"10.1038\/nrendo.2016.14","article-title":"Hypophosphatasia - aetiology, nosology, pathogenesis, diagnosis and treatment","volume":"12","author":"Whyte MP","year":"2016","unstructured":"Whyte MP. Hypophosphatasia - aetiology, nosology, pathogenesis, diagnosis and treatment. Nat Rev Endocrinol. 2016, 12:233-46. 10.1038\/nrendo.2016.14","journal-title":"Nat Rev Endocrinol"},{"key":"ref3","doi-asserted-by":"publisher","DOI":"10.1530\/boneabs.7.P114","article-title":"Hypophosphatasia in Japan: ALPL mutation analysis in 98 unrelated patients","volume":"106","author":"Michigami T","year":"2000","unstructured":"Michigami T, Tachikawa K, Yamazaki M, Kawai M, Kubota T, Ozono K. Hypophosphatasia in Japan: ALPL mutation analysis in 98 unrelated patients. Calcified Tissue Int. 2000, 106:221-31. 10.1530\/boneabs.7.P114","journal-title":"Calcified Tissue Int"},{"key":"ref4","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1106173","article-title":"Enzyme-replacement therapy in life-threatening hypophosphatasia","volume":"366","author":"Whyte MP","year":"2012","unstructured":"Whyte MP, Greenberg CR, Salman NJ, et al.. Enzyme-replacement therapy in life-threatening hypophosphatasia. N Engl J Med. 2012, 366:904-13. 10.1056\/NEJMoa1106173","journal-title":"N Engl J Med"},{"key":"ref5","doi-asserted-by":"publisher","DOI":"10.1210\/jc.2015-3462","article-title":"Asfotase alfa treatment improves survival for perinatal and infantile hypophosphatasia","volume":"101","author":"Whyte MP","year":"2016","unstructured":"Whyte MP, Rockman-Greenberg C, Ozono K, et al.. Asfotase alfa treatment improves survival for perinatal and infantile hypophosphatasia. J Clin Endocrinol Metab. 2016, 101:334-42. 10.1210\/jc.2015-3462","journal-title":"J Clin Endocrinol Metab"},{"key":"ref6","doi-asserted-by":"publisher","DOI":"10.1172\/jci.insight.85971","article-title":"Asfotase alfa therapy for children with hypophosphatasia","volume":"1","author":"Whyte MP","year":"2016","unstructured":"Whyte MP, Madson KL, Phillips D, et al.. Asfotase alfa therapy for children with hypophosphatasia. JCI Insight. 2016, 1:85971. 10.1172\/jci.insight.85971","journal-title":"JCI Insight"},{"key":"ref7","doi-asserted-by":"publisher","DOI":"10.1111\/cga.12374","article-title":"Perinatal benign hypophosphatasia antenatally diagnosed through measurements of parental serum alkaline phosphatase and ultrasonography","volume":"60","author":"Io S","year":"2020","unstructured":"Io S, Watanabe A, Yamada S, Mandai M, Yamada T. Perinatal benign hypophosphatasia antenatally diagnosed through measurements of parental serum alkaline phosphatase and ultrasonography. Congenit Anom (Kyoto). 2020, 60:199-200. 10.1111\/cga.12374","journal-title":"Congenit Anom (Kyoto)"},{"key":"ref8","doi-asserted-by":"publisher","DOI":"10.1297\/cpe.30.53","article-title":"Asfotase alfa has a limited effect in improving the bowed limbs in perinatal benign hypophosphatasia: a case report","volume":"30","author":"Matsushita M","year":"2021","unstructured":"Matsushita M, Mishima K, Nagata T, Kamiya Y, Imagama S, Kitoh H. Asfotase alfa has a limited effect in improving the bowed limbs in perinatal benign hypophosphatasia: a case report. Clin Pediatr Endocrinol. 2021, 30:53-6. 10.1297\/cpe.30.53","journal-title":"Clin Pediatr Endocrinol"},{"key":"ref9","doi-asserted-by":"publisher","DOI":"10.1007\/s00431-015-2641-2","article-title":"Lethal hypophosphatasia successfully treated with enzyme replacement from day 1 after birth","volume":"175","author":"Okazaki Y","year":"2016","unstructured":"Okazaki Y, Kitajima H, Mochizuki N, Kitaoka T, Michigami T, Ozono K. Lethal hypophosphatasia successfully treated with enzyme replacement from day 1 after birth. Eur J Pediatr. 2016, 175:433-7. 10.1007\/s00431-015-2641-2","journal-title":"Eur J Pediatr"},{"key":"ref10","doi-asserted-by":"publisher","DOI":"10.1002\/jcu.1870150305","article-title":"Ultrasonic mensuration of fetal limb bones in the second and third trimesters","volume":"15","author":"Merz E","year":"1987","unstructured":"Merz E, Kim-Kern MS, Pehl S. Ultrasonic mensuration of fetal limb bones in the second and third trimesters. J Clin Ultrasound. 1987, 15:175-83. 10.1002\/jcu.1870150305","journal-title":"J Clin Ultrasound"},{"key":"ref11","doi-asserted-by":"publisher","DOI":"10.1148\/radiology.177.1.2399317","article-title":"Usefulness of a short femur in the in utero detection of skeletal dysplasias","volume":"177","author":"Kurtz AB","year":"1990","unstructured":"Kurtz AB, Needleman L, Wapner RJ, et al.. Usefulness of a short femur in the in utero detection of skeletal dysplasias. Radiology. 1990, 177:197-200. 10.1148\/radiology.177.1.2399317","journal-title":"Radiology"},{"key":"ref12","doi-asserted-by":"publisher","DOI":"10.1007\/s00247-012-2381-7","article-title":"Prenatal diagnosis of fetal skeletal dysplasia with 3D CT","volume":"42","author":"Miyazaki O","year":"2012","unstructured":"Miyazaki O, Nishimura G, Sago H, Horiuchi T, Hayashi S, Kosaki R. Prenatal diagnosis of fetal skeletal dysplasia with 3D CT. Pediatr Radiol. 2012, 42:842-52. 10.1007\/s00247-012-2381-7","journal-title":"Pediatr Radiol"},{"key":"ref13","doi-asserted-by":"publisher","DOI":"10.1002\/uog.12298","article-title":"Three-dimensional helical computed tomography in prenatal diagnosis of fetal skeletal dysplasia","volume":"42","author":"Mac\u00e9 G","year":"2013","unstructured":"Mac\u00e9 G, Sonigo P, Cormier-Daire V, et al.. Three-dimensional helical computed tomography in prenatal diagnosis of fetal skeletal dysplasia. Ultrasound Obstet Gynecol. 2013, 42:161-8. 10.1002\/uog.12298","journal-title":"Ultrasound Obstet Gynecol"},{"key":"ref14","doi-asserted-by":"publisher","DOI":"10.1186\/s12891-020-03663-x","article-title":"Prenatal diagnosis of fetal skeletal dysplasia using 3-dimensional computed tomography: a prospective study","volume":"21","author":"Waratani M","year":"2020","unstructured":"Waratani M, Ito F, Tanaka Y, Mabuchi A, Mori T, Kitawaki J. Prenatal diagnosis of fetal skeletal dysplasia using 3-dimensional computed tomography: a prospective study. BMC Musculoskelet Disord. 2020, 21:662. 10.1186\/s12891-020-03663-x","journal-title":"BMC Musculoskelet Disord"},{"key":"ref15","doi-asserted-by":"publisher","DOI":"10.1136\/archdischild-2013-305037","article-title":"Clinical and genetic aspects of hypophosphatasia in Japanese patients","volume":"99","author":"Taketani T","year":"2014","unstructured":"Taketani T, Onigata K, Kobayashi H, Mushimoto Y, Fukuda S, Yamaguchi S. Clinical and genetic aspects of hypophosphatasia in Japanese patients. Arch Dis Child. 2014, 99:211-5. 10.1136\/archdischild-2013-305037","journal-title":"Arch Dis Child"}],"container-title":["Cureus"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.cureus.com\/articles\/334795-long-term-outcomes-of-early-enzyme-replacement-therapy-with-asfotase-alfa-in-perinatal-benign-hypophosphatasia-amelioration-of-bone-deformities-in-a-young-child","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,2,4]],"date-time":"2025-02-04T09:02:32Z","timestamp":1738659752000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.cureus.com\/articles\/334795-long-term-outcomes-of-early-enzyme-replacement-therapy-with-asfotase-alfa-in-perinatal-benign-hypophosphatasia-amelioration-of-bone-deformities-in-a-young-child"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,2,4]]},"references-count":15,"URL":"https:\/\/doi.org\/10.7759\/cureus.78473","relation":{},"ISSN":["2168-8184"],"issn-type":[{"value":"2168-8184","type":"print"}],"subject":[],"published":{"date-parts":[[2025,2,4]]}}}