Expert Opinion on Orphan Drugs

Papers
(The H4-Index of Expert Opinion on Orphan Drugs is 6. 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 2021-11-01 to 2025-11-01.)
ArticleCitations
Risdiplam as an orphan drug treatment of spinal muscular atrophy in adults and children (2 months or older)18
Quality of life of patients with acromegaly: comparison of different therapeutic modalities14
Making orphan drugs and services available and accessible for people who live with rare diseases: what has been done? a systematic scoping review13
Management of pulmonary hypertension in infants8
Molecular aspects of the altered Angiotensin II signaling in Gitelman’s syndrome8
Elucidating the pathogenesis of adenosine deaminase 2 deficiency: current status and unmet needs6
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