Expert Opinion on Orphan Drugs

Papers
(The TQCC of Expert Opinion on Orphan Drugs is 4. 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 2021-05-01 to 2025-05-01.)
ArticleCitations
Quality of life of patients with acromegaly: comparison of different therapeutic modalities10
Risdiplam as an orphan drug treatment of spinal muscular atrophy in adults and children (2 months or older)10
An evaluation of nifurtimox for Chagas disease in children10
Making orphan drugs and services available and accessible for people who live with rare diseases: what has been done? a systematic scoping review7
Molecular aspects of the altered Angiotensin II signaling in Gitelman’s syndrome6
Efficacy of sirolimus for treatment of autoimmune lymphoproliferative syndrome: a systematic review of open label clinical studies5
Overview of genetic testing in Prader-Willi syndrome4
Elucidating the pathogenesis of adenosine deaminase 2 deficiency: current status and unmet needs4
Management of pulmonary hypertension in infants4
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