Expert Opinion on Orphan Drugs

Papers
(The TQCC 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
Overview of genetic testing in Prader-Willi syndrome6
Elucidating the pathogenesis of adenosine deaminase 2 deficiency: current status and unmet needs6
Management of pulmonary hypertension in infants6
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