Synageva BioPharma (United States)

A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency

Barbara K. Burton, Manisha Balwani, François Feillet et al. · New England Journal of Medicine · 2015 · 238 citations · Full text

Clinical Effect And Safety Profile of Recombinant Human Lysosomal Acid Lipase in Patients With Cholesteryl Ester Storage Disease

Manisha Balwani, Catherine Breen, Gregory M. Enns et al. · Hepatology · 2013 · 116 citations · Full text

Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants

Simon Jones, Vassili Valayannopoulos, Eugene Schneider et al. · Genetics in Medicine · 2015 · 113 citations · Full text

Clinical Features of Lysosomal Acid Lipase Deficiency

Barbara K. Burton, Patrick Deegan, Gregory M. Enns et al. · Journal of Pediatric Gastroenterology and Nutrition · 2015 · 111 citations · Full text

Sebelipase alfa over 52weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency

Vassili Valayannopoulos, Vĕra Malinová, Tomáš Honzík et al. · Journal of Hepatology · 2014 · 95 citations · Full text