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Enzyme-replacement therapy in life-threatening hypophosphatasia.
N Engl J Med. 2012 Mar 8;366(10):904-13. doi: 10.1056/NEJMoa1106173.
N Engl J Med. 2012.
PMID: 22397652
Free article.
Clinical Trial.
Characterization of tracheobronchomalacia in infants with hypophosphatasia.
Padidela R, Yates R, Benscoter D, McPhail G, Chan E, Nichani J, Mughal MZ, Myer C 4th, Narayan O, Nissenbaum C, Wilkinson S, Zhou S, Saal HM.
Padidela R, et al.
Orphanet J Rare Dis. 2020 Aug 6;15(1):204. doi: 10.1186/s13023-020-01483-9.
Orphanet J Rare Dis. 2020.
PMID: 32762706
Free PMC article.
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Asfotase Alfa Treatment Improves Survival for Perinatal and Infantile Hypophosphatasia.
Whyte MP, Rockman-Greenberg C, Ozono K, Riese R, Moseley S, Melian A, Thompson DD, Bishop N, Hofmann C.
Whyte MP, et al.
J Clin Endocrinol Metab. 2016 Jan;101(1):334-42. doi: 10.1210/jc.2015-3462. Epub 2015 Nov 3.
J Clin Endocrinol Metab. 2016.
PMID: 26529632
Free PMC article.
Clinical Trial.
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