Cryopyrin-associated periodic syndrome
Interleukin-1β inhibitors for the treatment of cryopyrin-associated periodic syndrome
... Abstract: Cryopyrin-associated periodic syndrome (CAPS) comprises a group of rare, but severe, inherited autoinflammatory disorders associated with aberrant secretion of interleukin ...
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Successful Management of Cryopyrin-Associated Periodic Syndrome With Canakinumab in Infancy
... (CINCA) syndrome is a rare, early-onset autoin fl ammatory disorder and the most severe form of cryopyrin-associated periodic syndrome, which is associated with over- production ...
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Decreased quality of life and societal impact of cryopyrin-associated periodic syndrome treated with canakinumab: a questionnaire based cohort study
... Background: Cryopyrin-associated periodic syndrome (CAPS) is a rare disease. Knowledge on the quality of life (QoL) and the disease ’ s societal impact is limited. Canakinumab is used in ...
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Interleukin-1 antagonists in the treatment of autoinflammatory syndromes, including cryopyrin-associated periodic syndrome
... Abstract: Cryopyrin-associated periodic syndrome (CAPS) include a group of rare autoinflammatory disorders, the spectrum of which ranges from the mildest form, ie, familial cold ...
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Retrospective case series describing the efficacy, safety and cost-effectiveness of a vial-sharing programme for canakinumab treatment for paediatric patients with cryopyrin-associated periodic syndrome
... Fig. 1 Cryopyrin associated periodic syndrome disease activity score pre-and post canakinumab. The difference between CAPS DAS scores before and after canakinumab treatment was highly ...
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Obvious optic disc swelling in a patient with cryopyrin-associated periodic syndrome
... Abstract: Cryopyrin-associated periodic syndrome (CAPS) is a group of rare hereditary autoinflammatory diseases caused by mutations of the NLRP3 gene, and leads to excessive production of the ...
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Canakinumab (ACZ885, a fully human IgG1 anti IL 1β mAb) induces sustained remission in pediatric patients with cryopyrin associated periodic syndrome (CAPS)
... Introduction: Cryopyrin-associated periodic syndrome (CAPS) represents a spectrum of three auto-inflammatory syndromes, familial cold auto-inflammatory syndrome (FCAS), Muckle-Wells ...
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Sustained remission of symptoms and improved health related quality of life in patients with cryopyrin associated periodic syndrome treated with canakinumab: results of a double blind placebo controlled randomized withdrawal study
... CAPS: cryopyrin-associated periodic syndrome; CHQ-PF28: Child Health Questionnaire-Parent Form 28; CINCA: chronic infantile neurologic cutaneous and articular syndrome; CRP: C- reactive ...
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The schedule of administration of canakinumab in cryopyrin associated periodic syndrome is driven by the phenotype severity rather than the age
... In a recent trial the use of subcutaneous doses of 150 mg (or 2 mg/kg) of canakinumab every 8 weeks for 24 weeks was generally associated with complete control of clinical manifestations and laboratory parameters ...
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Critical appraisal of canakinumab in the treatment of adults and children with cryopyrin-associated periodic syndrome (CAPS)
... The cryopyrin-associated syndromes (CAPS) include three autosomal-dominant syndromes, that are caused by a mutation in the NLRP3 gene on chromosome 1, encoding the cryopyrin ...autoinflammatory ...
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Methodological challenges in monitoring new treatments for rare diseases: lessons from the cryopyrin-associated periodic syndrome registry
... Knowledge of the disease, although improving, is still limited. Disease symptoms generally appear in early childhood, but sensorineural deafness, one characteristic feature of MWS, develops in up to two-thirds of ...
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Neurological outcome of patients with cryopyrin-associated periodic syndrome (CAPS)
... Before any anti- IL-1β treatment, one patient had white matter lesions with demyelination as it may be seen in multiple sclerosis without any neurological sign (except for chronic headaches) unlike the only other CAPS ...
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Successful resolution of stromal keratitis and uveitis using canakinumab in a patient with chronic infantile neurologic, cutaneous, and articular syndrome: a case study
... Background: Cryopyrin-associated periodic syndrome (CAPS) is a group of rare autoinflammatory diseases, and of these, chronic infantile neurologic, cutaneous, and articular/neonatal-onset ...
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Tolerance and efficacy of off-label anti-interleukin-1 treatments in France: a nationwide survey
... (27), cryopyrin-associated periodic syndrome (CAPS) (21), familial Mediterranean fever (14) and mevalonate kinase deficiency ...s syndrome, gout, CAPS and ...significantly ...
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Comprehensive genetic testing for primary immunodeficiency disorders in a tertiary hospital: 10-year experience in Auckland, New Zealand
... uremic syndrome; ALPS autoimmune lymphoproliferative syndrome; APECED autoimmune polyendocrinopathy type 1; CGD-XL X-linked chronic granulomatous disease; CGD-AR autosomal recessive chronic granulomatous ...
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Muckle–Wells syndrome: clinical perspectives
... Abstract: Muckle–Wells syndrome (MWS) is a rare autoinflammatory disorder. It is due to NLRP3 gene mutations, responsible for excessive caspase-1 activation and interleukin 1 β processing. MWS is the intermediate ...
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Anakinra and related drugs targeting interleukin-1 in the treatment of cryopyrin- associated periodic syndromes
... articular syndrome, is the most severe clinical phenotype in the spec- trum of cryopyrin-associated periodic ...protein cryopyrin, leading to activation of the “NLRP3 inflammasome ...
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Monogenic autoinflammatory diseases in children: single center experience with clinical, genetic, and imaging review
... Aicardi-Goutieres syndrome; ARDS: Acute respiratory distress syndrome; CAPS: Cryopyrin- associated periodic syndromes; CINCA syndrome: Chronic infantile neurologic cutaneous and ...
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Autoinflammatory syndromes: diagnosis and management
... IgD syndrome (HIDS), tumor necro- sis factor receptor associated autoinflammatory syn- drome (TRAPS), cryopyrin associated periodic syndromes (CAPS), Blau syndrome and pyogenic ...
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CARD8 is a negative regulator for NLRP3 inflammasome, but mutant NLRP3 in cryopyrin associated periodic syndromes escapes the restriction
... autoinflammatory syndrome 1 ( CIAS1 ) gene encoding NLRP3 result in cryopyrin-associated periodic syndromes (CAPS) character- ized by recurrent episodes of systemic inflammatory attacks in the ...
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