Expert Opinion on Orphan Drugs

Papers
(The TQCC of Expert Opinion on Orphan Drugs is 3. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2021-04-01 to 2025-04-01.)
ArticleCitations
An evaluation of nifurtimox for Chagas disease in children10
Patient experience in a rare disease: application of the IEXPAC questionnaire in hereditary transthyretin-mediated amyloidosis10
Risdiplam as an orphan drug treatment of spinal muscular atrophy in adults and children (2 months or older)8
Pricing and reimbursement policy for new orphan drugs in South Korea: focused on patient accessibility and budget impact6
Recommendations for overcoming challenges in the diagnosis of lysosomal acid lipase deficiency5
The Orphan Drug Act and rare cancers: a retrospective analysis of oncologic orphan drug designations and associated approvals from 1983-20225
Pitolisant for the treatment of cataplexy in adults with narcolepsy4
Targeting the IL-2 pathway for the treatment of mucosal melanoma4
European stakeholder perspectives on challenges to rare disease drug development – a qualitative study3
Steroid alternatives for managing eosinophilic lung diseases3
Molecular aspects of the altered Angiotensin II signaling in Gitelman’s syndrome3
Advances in predicting patient survival in pulmonary sarcoidosis3
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