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 2020-03-01 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
Current state of developing advanced therapies for rare diseases in the European Union5
Steroid alternatives for managing eosinophilic lung diseases5
Advances in drug therapies for cytomegalovirus in transplantation: a focus on maribavir and letermovir5
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
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
Mexiletine (NaMuscla) for the treatment of myotonia in non-dystrophic myotonic disorders4
Gut microbial profiling as a therapeutic and diagnostic target for managing primary biliary cholangitis.3
Fusariosis: an update on therapeutic options for management3
Diagnostics and management approaches forAcanthamoebakeratitis3
Current and emerging treatment options to prevent renal failure due to autosomal dominant polycystic kidney disease3
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