Access to Orphan Drugs is a Challenge for Sustainable Management of Cystinosis in China
Author:
Publisher
Ovid Technologies (Wolters Kluwer Health)
Subject
General Medicine
Reference7 articles.
1. Cystinosis;Gahl;N Engl J Med,2002
2. Improved renal function in children with cystinosis treated with cysteamine;Markello;N Engl J Med,1993
3. Cystinosis: A review;Elmonem;Orphanet J Rare Dis,2016
4. Long-term treatment with growth hormone in short children with nephropathic cystinosis;Wühl;J Pediatr,2001
5. Rare diseases and legislation in China;Wang;Lancet,2010
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1. Worldwide disparities in access to treatment and investigations for nephropathic cystinosis: a 2023 perspective;Pediatric Nephrology;2023-11-18
2. Analysis on the Marketing Trend and Approval Lag of Imported Orphan Drugs from 2010 to 2021 in China;Therapeutic Innovation & Regulatory Science;2023-08-31
3. A systematic review of moral reasons on orphan drug reimbursement;Orphanet Journal of Rare Diseases;2021-06-30
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