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- W2021193835 abstract "Cryopyrin-associated periodic fever syndrome (CAPS) is a rare inherited disease due to mutations in the NLRP3 (also called CIAS1) gene on chromosome 1q44 resulting in overproduction of interleukin-1. CAPS comprises three clinically overlapping disorders including Muckle-Wells syndrome. We report on two half-siblings with Muckle-Wells syndrome who were successfully treated with the interleukin-1 beta antibody canakinumab. Despite reduced dosing and longer treatment intervals compared to the recommended dosing schedule (e.g. 150 mg every 8 weeks), the efficacy and tolerability of canakinumab was impressive in both patients. The pharmacologic properties of canakinumab are reviewed and the clinical and economical aspects highlighted. We show that with individualized 'reflare-guided' administrations of canakinumab overall costs could hypothetically be reduced by 50% (approx. USD 60,000/patient/year) and therefore could have a major impact on treatment costs." @default.
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- W2021193835 date "2011-01-01" @default.
- W2021193835 modified "2023-09-25" @default.
- W2021193835 title "Muckle-Wells Syndrome Effectively Treated with Canakinumab: Is the Recommended Dosing Schedule Mandatory?" @default.
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- W2021193835 doi "https://doi.org/10.1159/000331580" @default.
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