Efficacy and Safety of Encaleret in Autosomal Dominant Hypocalcemia Type 1.
作者信息
Gafni Rachel I, Hartley Iris R, Roszko Kelly L, Nemeth Edward F, Pozo Karen A, Lombardi Ed, Sridhar Ananth V, Roberts Mary S, Fox Jonathan C, Collins Michael T
机构信息
National Institutes of Health, Bethesda, MD
MetisMedica, Toronto, ON, Canada.
出版信息
N Engl J Med. 2023 Sep 28;389(13):1245-1247. doi: 10.1056/NEJMc2302708.
Abstract
摘要
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本文引用的文献
1
Autosomal Dominant Hypocalcemia Type 1: A Systematic Review.
J Bone Miner Res. 2022 Oct;37(10):1926-1935. doi: 10.1002/jbmr.4659. Epub 2022 Aug 22.
2
Treatment of Autosomal Dominant Hypocalcemia Type 1 With the Calcilytic NPSP795 (SHP635).
J Bone Miner Res. 2019 Sep;34(9):1609-1618. doi: 10.1002/jbmr.3747. Epub 2019 Jul 26.
3
Calcilytic Ameliorates Abnormalities of Mutant Calcium-Sensing Receptor (CaSR) Knock-In Mice Mimicking Autosomal Dominant Hypocalcemia (ADH).
J Bone Miner Res. 2015 Nov;30(11):1980-93. doi: 10.1002/jbmr.2551. Epub 2015 Jul 16.
4
Autosomal dominant hypocalcaemia caused by a Ca(2+)-sensing receptor gene mutation.
Nat Genet. 1994 Nov;8(3):303-7. doi: 10.1038/ng1194-303.
5
Calcium-ion-sensing cell-surface receptors.
N Engl J Med. 1995 Jul 27;333(4):234-40. doi: 10.1056/NEJM199507273330407.
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