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 2021-08-01 to 2025-08-01.)
ArticleCitations
Risdiplam as an orphan drug treatment of spinal muscular atrophy in adults and children (2 months or older)14
Quality of life of patients with acromegaly: comparison of different therapeutic modalities12
Making orphan drugs and services available and accessible for people who live with rare diseases: what has been done? a systematic scoping review11
Molecular aspects of the altered Angiotensin II signaling in Gitelman’s syndrome10
Efficacy of sirolimus for treatment of autoimmune lymphoproliferative syndrome: a systematic review of open label clinical studies7
Management of pulmonary hypertension in infants6
Overview of genetic testing in Prader-Willi syndrome6
Elucidating the pathogenesis of adenosine deaminase 2 deficiency: current status and unmet needs6
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