PubMed · 2013 · 13 citations · 0 references
Life SciencesGeneticsPediatricsHuman GeneticsMedical GeneticsPediatric OtolaryngologyCongenital DisordersGenetic DisordersGenetic SyndromesHearing ImpairmentHearing LossRare Disease GeneticsAutosomal Dominant InheritanceChildhood Sensorineural Hearing LossGenetic Inner Ear Disorders
Muckle-Wells syndrome (MWS) is a rare autosomal dominant condition with variable expression. It is a subset of auto-inflammatory diseases characterised by recurrent inflammatory crises and is associated with chronic recurrent urticaria, sensorineural deafness, periodic arthritis and secondary amyloidosis. The diagnosis of MWS is a clinical one with sufferers classically presenting in childhood with a moderate fever and non-pruiginous urticaria. We describe a case of a six-year-old girl who was successfully diagnosed and treated with Anakinra. Muckle and Wells originally described this syndrome in 1962; however, only recently was it discovered to be genetically linked to chromosome 1q44 and subsequently to missense mutations in the CIAS1/NALP3/PYPAF1 gene. Since then, treatment has evolved and it remains one of few treatable causes of congenital profound sensorineural hearing loss.