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Asfotase alfa treatment in perinatal and infantile hypophosphatasia: safe and sustained efficacy.

作者信息

Padidela Raja

机构信息

Paediatric Endocrinology and Metabolic Bone Disorders, Royal Manchester Children's Hospital, Manchester, UK; Faculty of Biology, Medicine, and Health, University of Manchester, Manchester M139WL, UK.

出版信息

Lancet Diabetes Endocrinol. 2019 Feb;7(2):76-78. doi: 10.1016/S2213-8587(18)30321-8. Epub 2018 Dec 14.

DOI:10.1016/S2213-8587(18)30321-8
PMID:30558908
Abstract
摘要

相似文献

1
Asfotase alfa treatment in perinatal and infantile hypophosphatasia: safe and sustained efficacy.阿法骨化醇治疗围产期和婴儿期低磷性佝偻病:安全性与持续疗效
Lancet Diabetes Endocrinol. 2019 Feb;7(2):76-78. doi: 10.1016/S2213-8587(18)30321-8. Epub 2018 Dec 14.
2
Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial.阿法磷酸酶治疗婴儿和幼儿低磷酸酯酶症:一项单臂、开放标签、2 期扩展试验的 7 年结果。
Lancet Diabetes Endocrinol. 2019 Feb;7(2):93-105. doi: 10.1016/S2213-8587(18)30307-3. Epub 2018 Dec 14.
3
Asfotase Alfa Treatment Improves Survival for Perinatal and Infantile Hypophosphatasia.阿法骨化醇治疗可提高围产期和婴儿期低磷酸酯酶症的生存率。
J Clin Endocrinol Metab. 2016 Jan;101(1):334-42. doi: 10.1210/jc.2015-3462. Epub 2015 Nov 3.
4
Safety and efficacy of treatment with asfotase alfa in patients with hypophosphatasia: Results from a Japanese clinical trial.低磷酸酯酶症患者使用阿法骨化醇治疗的安全性和有效性:一项日本临床试验的结果。
Clin Endocrinol (Oxf). 2017 Jul;87(1):10-19. doi: 10.1111/cen.13343. Epub 2017 May 2.
5
Asfotase Alfa in Perinatal/Infantile-Onset and Juvenile-Onset Hypophosphatasia: A Guide to Its Use in the USA.阿法骨化醇酶在围产期/婴儿期发病型和青少年期发病型低磷性骨软化症中的应用:美国使用指南
BioDrugs. 2016 Feb;30(1):41-8. doi: 10.1007/s40259-016-0161-x.
6
Dental outcomes for children receiving asfotase alfa for hypophosphatasia.接受阿法骨化醇治疗低磷性佝偻病的儿童的牙齿预后。
Bone. 2021 Nov;152:116089. doi: 10.1016/j.bone.2021.116089. Epub 2021 Jun 25.
7
Letter to the Editor: "Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study".致编辑的信:“阿法骨化醇酶在低磷血症婴幼儿中的疗效和安全性:一项2期开放标签研究”
J Clin Endocrinol Metab. 2019 Aug 1;104(8):3146-3147. doi: 10.1210/jc.2018-02413.
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Asfotase alfa therapy for children with hypophosphatasia.阿法磷酸酶治疗低磷酸酶血症儿童。
JCI Insight. 2016 Jun 16;1(9):e85971. doi: 10.1172/jci.insight.85971.
9
Case Report: Efficacy of Reduced Doses of Asfotase Alfa Replacement Therapy in an Infant With Hypophosphatasia Who Lacked Severe Clinical Symptoms.病例报告:在一名缺乏严重临床症状的低磷酸酶血症婴儿中,减少阿法特酶替代治疗剂量的疗效。
Front Endocrinol (Lausanne). 2020 Dec 18;11:590455. doi: 10.3389/fendo.2020.590455. eCollection 2020.
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Asfotase Alfa: A Review in Paediatric-Onset Hypophosphatasia.阿法磷酸酶:儿科发病低磷酸酯酶症的综述。
Drugs. 2016 Feb;76(2):255-62. doi: 10.1007/s40265-015-0535-2.

引用本文的文献

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Effects of asfotase alfa on fracture healing of adult patient with hypophosphatasia and literature review.阿法骨化醇对低磷性骨软化症成年患者骨折愈合的影响及文献综述
Orphanet J Rare Dis. 2025 Apr 6;20(1):162. doi: 10.1186/s13023-025-03663-x.
2
The impact of enzyme replacement therapy on the oral health manifestations of hypophosphatasia among children: a scoping review.酶替代疗法对儿童低磷酸酯酶症口腔健康表现的影响:范围综述。
Eur Arch Paediatr Dent. 2023 Aug;24(4):429-440. doi: 10.1007/s40368-023-00796-0. Epub 2023 Apr 10.
3
Deletion of the Pyrophosphate Generating Enzyme ENPP1 Rescues Craniofacial Abnormalities in the TNAP Mouse Model of Hypophosphatasia and Reveals FGF23 as a Marker of Phenotype Severity.
焦磷酸生成酶ENPP1的缺失挽救了低磷酸酯酶症TNAP小鼠模型中的颅面异常,并揭示FGF23作为表型严重程度的标志物。
Front Dent Med. 2022;3. doi: 10.3389/fdmed.2022.846962. Epub 2022 Apr 28.
4
Hypophosphatasia: Biological and Clinical Aspects, Avenues for Therapy.低磷酸酯酶症:生物学与临床方面及治疗途径
Clin Biochem Rev. 2020 Feb;41(1):13-27. doi: 10.33176/AACB-19-00031.