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Biotech / Medical : Alexion Pharmaceuticals, Inc. (ALXN)

ALXN 182.50

0.0%

Jul 28 4:00 PM EDT

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From: idos	1/29/2009 6:36:51 AM
	of 824

Two papers from the NEJM (thanks to David Miller who doesn't like the journal and made me look at the table of contents) Eculizumab for Atypical Hemolytic–Uremic Syndrome content.nejm.org To the Editor: Atypical hemolytic–uremic syndrome is a disease of uncontrolled complement activation associated with a high mortality rate, and most cases progress to end-stage renal disease.1 About 50% of patients with this syndrome carry mutations in genes encoding complement proteins.2 Complement inhibition has been suggested for the treatment of atypical hemolytic–uremic syndrome,3 but currently no data on this treatment option are available. We report on a case of atypical hemolytic–uremic syndrome that was successfully treated with eculizumab, a humanized monoclonal antibody that blocks complement activity by cleavage of the complement protein C5, thereby preventing the generation of the inflammatory . . . Eculizumab for Congenital Atypical Hemolytic–Uremic Syndrome content.nejm.org To the Editor: Atypical hemolytic–uremic syndrome of infancy is a rare disorder that is associated with thrombotic microangiopathy and acute renal failure. It often involves complement dysregulation.1,2 Plasma infusions have variable efficacy, and end-stage renal disease often develops in children who are unresponsive to plasma therapy.1,2 We report on a patient with congenital relapsing atypical hemolytic–uremic syndrome who was unresponsive to plasma therapy but had a response to eculizumab, a humanized monoclonal antibody against terminal complement protein C5.3 An 18-month-old boy was admitted with a fourth relapse of congenital atypical hemolytic–uremic syndrome. He was born at 34 weeks' gestation, and . . .

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