OOIR: Observatory of International Research

Papers

(The median citation count of Expert Opinion on Orphan Drugs is 1. 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 2021-02-01 to 2025-02-01.)

Article	Citations
An evaluation of nifurtimox for Chagas disease in children	10
Canakinumab injection for the treatment of active Still’s disease, including adult-onset Still’s disease	8
Patient experience in a rare disease: application of the IEXPAC questionnaire in hereditary transthyretin-mediated amyloidosis	8
Risdiplam as an orphan drug treatment of spinal muscular atrophy in adults and children (2 months or older)	6
Recommendations for overcoming challenges in the diagnosis of lysosomal acid lipase deficiency	4
Pricing and reimbursement policy for new orphan drugs in South Korea: focused on patient accessibility and budget impact	4
Targeting the IL-2 pathway for the treatment of mucosal melanoma	4
A review of the criteria for non-invasive diagnosis of cardiac transthyretin amyloidosis	4
Pitolisant for the treatment of cataplexy in adults with narcolepsy	3
Advances in predicting patient survival in pulmonary sarcoidosis	3
Steroid alternatives for managing eosinophilic lung diseases	3
Hydrocortisone granules in capsules for opening (Alkindi) as replacement therapy in pediatric patients with adrenal insufficiency	3
The Orphan Drug Act and rare cancers: a retrospective analysis of oncologic orphan drug designations and associated approvals from 1983-2022	3
Orphan drugs approved in Canada: health technology assessment, price negotiation, and government formulary listing	2
Quality of life of patients with acromegaly: comparison of different therapeutic modalities	2
Making orphan drugs and services available and accessible for people who live with rare diseases: what has been done? a systematic scoping review	2
European stakeholder perspectives on challenges to rare disease drug development – a qualitative study	2
Assessing and managing symptom burden and quality of life in primary sclerosing cholangitis patients	2
Molecular aspects of the altered Angiotensin II signaling in Gitelman’s syndrome	2
Elucidating the pathogenesis of adenosine deaminase 2 deficiency: current status and unmet needs	1
Real world data for rare diseases research: The beginner’s guide to registries	1
Emerging therapies against Naegleria fowleri	1
Old and novel prognostic biomarkers in primary biliary cholangitis	1