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BioWorld - Friday, April 24, 2026
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In the clinic

Nov. 6, 2017
Alexion Pharmaceuticals Inc., of New Haven, Conn., reported new interim data from a trial testing Kanuma (sebelipase alfa) in patients with rapidly progressive lysosomal acid lipase deficiency at the NASPGHAN Annual Meeting in Las Vegas. Of the 10 infants treated with Kanuma, nine survived beyond 1 year of age, compared to a median age of death of 3.7 months with mortality by 1 year of nearly 100 percent for historically untreated infants.
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