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Current and Future Therapeutical Aspects of X-Linked Hypophosphatemia in Children With Special Attention to Klotho/Fibroblast Growth Factor 23 System.

作者信息

Bittmann Stefan, Moschuring-Alieva Elena, Luchter Elisabeth, Villalon Gloria

机构信息

Department of Pediatrics, Ped Mind Institute, Hindenburgring 4, D-48599 Gronau, Germany.

出版信息

J Clin Med Res. 2022 Oct;14(10):436-439. doi: 10.14740/jocmr4820. Epub 2022 Oct 28.

DOI:10.14740/jocmr4820
PMID:36406946
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9635803/
Abstract
摘要

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Current and Future Therapeutical Aspects of X-Linked Hypophosphatemia in Children With Special Attention to Klotho/Fibroblast Growth Factor 23 System.X连锁低磷血症在儿童中的当前及未来治疗方面,特别关注Klotho/成纤维细胞生长因子23系统
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2
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本文引用的文献

1
A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia.一种治疗骨骼疾病的新策略:X连锁低磷血症基因治疗的概念验证。
Sci Adv. 2021 Oct 29;7(44):eabj5018. doi: 10.1126/sciadv.abj5018. Epub 2021 Oct 27.
2
Sustained Efficacy and Safety of Burosumab, a Monoclonal Antibody to FGF23, in Children With X-Linked Hypophosphatemia.Burosumab,一种成纤维细胞生长因子 23 的单克隆抗体,在 X 连锁低磷血症儿童中的持续疗效和安全性。
J Clin Endocrinol Metab. 2022 Feb 17;107(3):813-824. doi: 10.1210/clinem/dgab729.
3
X-Linked Hypophosphatemic Rickets: Multisystemic Disorder in Children Requiring Multidisciplinary Management.X 连锁低磷血症性佝偻病:需要多学科管理的儿童多系统疾病。
Front Endocrinol (Lausanne). 2021 Aug 6;12:688309. doi: 10.3389/fendo.2021.688309. eCollection 2021.
4
Monitoring response to conventional treatment in children with XLH: Value of ALP and Rickets Severity Score (RSS) in a real world setting.监测 XLH 患儿对常规治疗的反应:真实世界环境中 ALP 和佝偻病严重程度评分(RSS)的价值。
Bone. 2021 Oct;151:116025. doi: 10.1016/j.bone.2021.116025. Epub 2021 May 28.
5
Burosumab for Pediatric X-Linked Hypophosphatemia.布罗索尤单抗治疗儿童 X 连锁低磷血症。
Curr Osteoporos Rep. 2021 Jun;19(3):271-277. doi: 10.1007/s11914-021-00669-9. Epub 2021 May 10.
6
Clinical guidelines for burosumab in the treatment of XLH in children and adolescents: British paediatric and adolescent bone group recommendations.布罗索尤单抗治疗儿童和青少年XLH的临床指南:英国儿科和青少年骨病组建议
Endocr Connect. 2020 Oct;9(10):1051-1056. doi: 10.1530/EC-20-0291.
7
Validation of a next-generation sequencing (NGS) panel to improve the diagnosis of X-linked hypophosphataemia (XLH) and other genetic disorders of renal phosphate wasting.下一代测序(NGS)panel 的验证可提高 X 连锁低磷血症(XLH)和其他遗传性磷丢失性肾病的诊断。
Eur J Endocrinol. 2020 Nov;183(5):497-504. doi: 10.1530/EJE-20-0275.
8
Burosumab in X-linked hypophosphatemia and perspective for chronic kidney disease.布罗索尤单抗治疗 X 连锁低磷血症及慢性肾脏病前景。
Curr Opin Nephrol Hypertens. 2020 Sep;29(5):531-536. doi: 10.1097/MNH.0000000000000631.
9
The international X-linked hypophosphataemia (XLH) registry (NCT03193476): rationale for and description of an international, observational study.国际 X 连锁低磷血症(XLH)登记处(NCT03193476):一项国际观察性研究的原理和描述。
Orphanet J Rare Dis. 2020 Jun 30;15(1):172. doi: 10.1186/s13023-020-01434-4.
10
Clinical Evidence for the Benefits of Burosumab Therapy for X-Linked Hypophosphatemia (XLH) and Other Conditions in Adults and Children.成人和儿童 X 连锁低磷血症(XLH)及其他疾病中使用布罗索尤单抗治疗的临床证据。
Front Endocrinol (Lausanne). 2020 May 28;11:338. doi: 10.3389/fendo.2020.00338. eCollection 2020.