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Adult onset still's disease (AOSD) is an autoinflammatory condition characterized by fevers, arthritis, and rash. It is considered an orphan disease because of its low prevalence - it is reported anywhere between 16 to 40 per ten million. Traditionally, a bimodal distribution by age has been
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Orphan drugs target rare dz <200k. New drugs (2008-2016) compared 5 yr sales 86 orphan Rx to 232 non-orphan = similar sales- Orphan ($719 mill) vs nonorphan ($812 mill). But orphan Rx lower volume offset by higher prices (7X higher) than nonorphan drugs https://t.co/I8kb4A0eyG https://t.co/gEQpssRVkg
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Anakinra Phase 2/3 study in severe hosp. COVID-19 pts (5/20-3/21) w/ pneumonia & hyperinflammation (incr levels IL-6, ferritin, CRP, LDH). 179 pt Rx w/ ANA or SOC - d15 need for Mech ventilation was 77% ANAK v 86% SoC (NS; RR 0.90; 0.77-1.04) https://t.co/CFcI1qTsU2 https://t.co/wAj4pHN5Dh
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LAVLI - A New Autoinflammatory Disorder NIH researchers have have described a novel autoinflammatory disorder called "Lyn kinase-associated vasculopathy and liver fibrosis" (LAVLI), based on a mutation in the LYN gene. https://t.co/RYmb3QFB1T https://t.co/sn5hwVxwPk
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LAVLI - A New Autoinflammatory Disorder NIH researchers have have described a novel autoinflammatory disorder called "Lyn kinase-associated vasculopathy and liver fibrosis" (LAVLI), based on a mutation in the LYN gene. https://t.co/VWs0YuF4LD https://t.co/7ydA3bb82j
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NIH researchers have have described a novel autoinflammatory disorder called "Lyn kinase-associated vasculopathy and liver fibrosis" (LAVLI), based on a mutation in the LYN gene (that encodes the Lyn kinase protein). They discovered that increased Lyn kinase activity promotes systemic
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