Access to Orphan Drugs is a Challenge for Sustainable Management of Cystinosis in China.
作者信息
Li Xiao-Qiao, Peng Xiao-Xia, Gong Chun-Xiu
机构信息
Beijing Key Laboratory for Genetics of Birth Defects, Center of Endocrinology, Genetics and Metabolism, Beijing Children's Hospital, The Capital Medical University, National Center for Children's Health, Beijing 100045, China.
Center of Clinical Epidemiology and Evidence-Based Medicine, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health; Department of Epidemiology and Biostatistics, School of Public Health, Capital Medical University, Beijing 100045, China.
出版信息
Chin Med J (Engl). 2018 Oct 5;131(19):2388-2389. doi: 10.4103/0366-6999.241814.
Abstract
摘要
相似文献
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Access to Orphan Drugs is a Challenge for Sustainable Management of Cystinosis in China.在中国，获取孤儿药是胱氨酸病可持续管理面临的一项挑战。
Chin Med J (Engl). 2018 Oct 5;131(19):2388-2389. doi: 10.4103/0366-6999.241814.
2
Cysteamine for cystinosis.用于胱氨酸病的半胱胺
Med Lett Drugs Ther. 1994 Dec 23;36(938):118.
3
Therapy of cystinosis.胱氨酸病的治疗
Prog Clin Biol Res. 1985;197:157-62.
4
A cost-effectiveness analysis of the orphan drug cysteamine in the treatment of infantile cystinosis.孤儿药半胱胺治疗婴儿胱氨酸病的成本效益分析。
Med Decis Making. 1997 Apr-Jun;17(2):193-8. doi: 10.1177/0272989X9701700210.
5
Approval of cysteamine for patients with cystinosis.半胱胺用于胱氨酸病患者的批准。
Pediatr Nephrol. 1995 Apr;9(2):254. doi: 10.1007/BF00860767.
6
Nephropathic cystinosis: effect of long-term cysteamine therapy.肾性胱氨酸病：长期半胱胺治疗的效果
Clin Nephrol. 1987 Jun;27(6):309-12.
7
[The treatment of cystinosis with cysteamine].[用半胱胺治疗胱氨酸贮积症]
Ann Pediatr (Paris). 1991 Jun;38(6):440.
8
Treatment of cystinosis with cysteamine.用半胱胺治疗胱氨酸病。
J Pediatr. 1979 May;94(5):838-40. doi: 10.1016/s0022-3476(79)80174-2.
9
Cysteamine treatment of nephropathic cystinosis.半胱胺治疗肾病性胱氨酸病。
Prog Clin Biol Res. 1983;127:125-31.
10
[Treatment of cystinosis with cysteamine].[用半胱胺治疗胱氨酸病]
Presse Med. 1983 Jan 22;12(3):167-8.
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A systematic review of moral reasons on orphan drug reimbursement.孤儿药补偿的道德理由系统评价。
Orphanet J Rare Dis. 2021 Jun 30;16(1):292. doi: 10.1186/s13023-021-01925-y.
本文引用的文献
1
Effects of long-term cysteamine treatment in patients with cystinosis.胱氨酸病患者长期半胱胺治疗的效果。
Pediatr Nephrol. 2019 Apr;34(4):571-578. doi: 10.1007/s00467-017-3856-4. Epub 2017 Dec 19.
2
Cystinosis: a review.胱氨酸病：综述
Orphanet J Rare Dis. 2016 Apr 22;11:47. doi: 10.1186/s13023-016-0426-y.
3
Rare disease patients in China anticipate the sunlight of legislation.中国的罕见病患者期待立法的曙光。
Drug Discov Ther. 2013 Jun;7(3):126-8.
4
China's first rare-disease registry is under development.中国首个罕见病登记系统正在建设中。
Lancet. 2011 Aug 27;378(9793):769-70. doi: 10.1016/S0140-6736(11)61375-5.
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Rare diseases and legislation in China.中国的罕见病与立法
Lancet. 2010 Feb 27;375(9716):708-9. doi: 10.1016/S0140-6736(10)60240-1.
6
Cystinosis.胱氨酸病
N Engl J Med. 2002 Jul 11;347(2):111-21. doi: 10.1056/NEJMra020552.
7
Long-term treatment with growth hormone in short children with nephropathic cystinosis.生长激素对患有肾病性胱氨酸病的矮小儿童的长期治疗。
J Pediatr. 2001 Jun;138(6):880-7. doi: 10.1067/mpd.2001.113263.
8
Improved renal function in children with cystinosis treated with cysteamine.用半胱胺治疗的胱氨酸病患儿肾功能改善。
N Engl J Med. 1993 Apr 22;328(16):1157-62. doi: 10.1056/NEJM199304223281604.
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