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Access to Orphan Drugs is a Challenge for Sustainable Management of Cystinosis in China.
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Cysteamine for cystinosis.
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Therapy of cystinosis.
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A cost-effectiveness analysis of the orphan drug cysteamine in the treatment of infantile cystinosis.
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Approval of cysteamine for patients with cystinosis.
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[The treatment of cystinosis with cysteamine].
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Treatment of cystinosis with cysteamine.
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Cysteamine treatment of nephropathic cystinosis.
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[Treatment of cystinosis with cysteamine].
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Worldwide disparities in access to treatment and investigations for nephropathic cystinosis: a 2023 perspective.
Pediatr Nephrol. 2024 Apr;39(4):1113-1123. doi: 10.1007/s00467-023-06179-3. Epub 2023 Nov 18.
2
Analysis on the Marketing Trend and Approval Lag of Imported Orphan Drugs from 2010 to 2021 in China.
Ther Innov Regul Sci. 2023 Nov;57(6):1314-1321. doi: 10.1007/s43441-023-00572-8. Epub 2023 Aug 31.
3
A systematic review of moral reasons on orphan drug reimbursement.
Orphanet J Rare Dis. 2021 Jun 30;16(1):292. doi: 10.1186/s13023-021-01925-y.

本文引用的文献

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Effects of long-term cysteamine treatment in patients with cystinosis.
Pediatr Nephrol. 2019 Apr;34(4):571-578. doi: 10.1007/s00467-017-3856-4. Epub 2017 Dec 19.
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Cystinosis: a review.
Orphanet J Rare Dis. 2016 Apr 22;11:47. doi: 10.1186/s13023-016-0426-y.
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China's first rare-disease registry is under development.
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Rare diseases and legislation in China.
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Cystinosis.
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Long-term treatment with growth hormone in short children with nephropathic cystinosis.
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Improved renal function in children with cystinosis treated with cysteamine.
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