Expert Opinion on Orphan Drugs

Papers
(The median citation count of Expert Opinion on Orphan Drugs is 2. 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 2021-12-01 to 2025-12-01.)
ArticleCitations
Risdiplam as an orphan drug treatment of spinal muscular atrophy in adults and children (2 months or older)20
Quality of life of patients with acromegaly: comparison of different therapeutic modalities15
Molecular aspects of the altered Angiotensin II signaling in Gitelman’s syndrome13
Making orphan drugs and services available and accessible for people who live with rare diseases: what has been done? a systematic scoping review9
Management of pulmonary hypertension in infants8
Elucidating the pathogenesis of adenosine deaminase 2 deficiency: current status and unmet needs6
Targeting the IL-2 pathway for the treatment of mucosal melanoma4
Overview of genetic testing in Prader-Willi syndrome4
Pricing and reimbursement policy for new orphan drugs in South Korea: focused on patient accessibility and budget impact3
Current and emerging therapies for the treatment of leishmaniasis2
Recommendations for the management of diarrhea with trofinetide use in Rett syndrome2
Recommendations for overcoming challenges in the diagnosis of lysosomal acid lipase deficiency2
Real world data for rare diseases research: The beginner’s guide to registries2
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