Expert Opinion on Orphan Drugs

Papers
(The TQCC of Expert Opinion on Orphan Drugs is 3. 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 2020-11-01 to 2024-11-01.)
ArticleCitations
Advances in understanding of Netherton syndrome and therapeutic implications25
Drugs and biologics receiving FDA orphan drug designation: an analysis of the most frequently designated products and their repositioning strategies10
Recommendations for the management of diarrhea with trofinetide use in Rett syndrome8
Lumasiran: expanding the treatment options for patients with primary hyperoxaluria type 18
Steroid alternatives for managing eosinophilic lung diseases6
Current and innovative therapeutic strategies for the treatment of giant cell arteritis4
Hydrocortisone granules in capsules for opening (Alkindi) as replacement therapy in pediatric patients with adrenal insufficiency4
Gut microbial profiling as a therapeutic and diagnostic target for managing primary biliary cholangitis.4
Coenzyme Q10 and the exclusive club of diseases that show a limited response to treatment4
Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?4
Elucidating the pathogenesis of adenosine deaminase 2 deficiency: current status and unmet needs3
Making orphan drugs and services available and accessible for people who live with rare diseases: what has been done? a systematic scoping review3
The national drug formulary listing process for orphan drugs in South Korea: narrative review focused on pricing and reimbursement pathways3
A review of the criteria for non-invasive diagnosis of cardiac transthyretin amyloidosis3
Current medicines hold promise in the treatment of orphan infections due to brain-eating amoebae3
Fusariosis: an update on therapeutic options for management3
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