Expert Opinion on Orphan Drugs

Papers
(The H4-Index of Expert Opinion on Orphan Drugs is 7. 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 2020-03-01 to 2024-03-01.)
ArticleCitations
Monitoring progression of retinitis pigmentosa: current recommendations and recent advances27
Advances in understanding of Netherton syndrome and therapeutic implications23
The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism11
Druggable targets, clinical trial design and proposed pharmacological management in fibrodysplasia ossificans progressiva11
Balamuthia mandrillaris: pathogenesis, diagnosis, and treatment8
Drugs and biologics receiving FDA orphan drug designation: an analysis of the most frequently designated products and their repositioning strategies8
Lumasiran: expanding the treatment options for patients with primary hyperoxaluria type 17
Advances in therapeutic treatment options for ANCA-associated vasculitis7
Tazemetostat as a treatment for epithelioid sarcoma7
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