Article
Therapy with eculizumab for patients with CD59 p.Cys89Tyr mutation.
Annals of neurology - 1 Nov 2016
Mevorach Dror, Reiner Inna, Grau Amir, Ilan Uri, Berkun Yackov, Ta-Shma Asaf, Elpeleg Orly, Shorer Zamir, Edvardson Simon, Tabib Adi
Abstract excerpt
OBJECTIVE: The objective of this work was to report on the outcome of eculizumab treatment in pediatric patients with recurrent acute predominantly motor, demyelinating neuropathy with conduction block, and chronic hemolysis attributed to p.Cys89Tyr mutation in the CD59 gene. METHODS: Four patients were recruited from our new registry of patients with homozygosity for the p.Cys89Tyr mutation on CD59. Participants...
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