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Navigating challenges and opportunities in orphan medicines: A spotlight commentary on rare diseases.

作者信息

Belančić Andrej, Gkrinia Elvira Meni Maria, Likić Robert, Vitezić Dinko

机构信息

Department of Basic and Clinical Pharmacology with Toxicology, University of Rijeka, Faculty of Medicine, Rijeka, Croatia.

Independent Researcher, Athens, Greece.

出版信息

Br J Clin Pharmacol. 2025 Apr;91(4):1084-1087. doi: 10.1002/bcp.70013. Epub 2025 Feb 16.

DOI:10.1002/bcp.70013
PMID:39956537
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11992654/
Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6ac4/11992654/3e1270a86fee/BCP-91-1084-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6ac4/11992654/3e1270a86fee/BCP-91-1084-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6ac4/11992654/3e1270a86fee/BCP-91-1084-g001.jpg

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Riding the gene therapy wave: challenges and opportunities for rare disease patients and clinicians.乘着基因治疗的浪潮：罕见病患者和临床医生的挑战与机遇。
Curr Opin Pediatr. 2024 Dec 1;36(6):619-623. doi: 10.1097/MOP.0000000000001402. Epub 2024 Sep 17.
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The landscape for rare diseases in 2024.
2024年罕见病的概况。
Lancet Glob Health. 2024 Mar;12(3):e341. doi: 10.1016/S2214-109X(24)00056-1.
4
An Ethics Action Plan for Rare Disease Care: Participatory Action Research Approach.罕见病护理伦理行动计划：参与式行动研究方法
J Particip Med. 2023 Nov 23;15:e46607. doi: 10.2196/46607.
5
Regulatory Considerations Toward Orphan Drug Designation and Orphan Drug Exclusivity in the United States and European Union: Structural Similarity, Clinical Superiority/Significant Benefit, and Case Studies.美国和欧盟对孤儿药指定及孤儿药独占权的监管考量：结构相似性、临床优势/显著获益及案例研究
Ther Innov Regul Sci. 2023 Mar;57(2):386-395. doi: 10.1007/s43441-022-00477-y. Epub 2022 Nov 18.
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Addressing the challenges of novel therapies in rare diseases with mechanistic perspectives: Missed opportunities or the way forward?从机制角度应对罕见病新疗法的挑战：错失的机遇还是前进的方向？
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Genome Med. 2022 Feb 28;14(1):23. doi: 10.1186/s13073-022-01026-w.
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