Sebelipase Alfa: First Global Approval

P1212 : Impact of sebelipase alfa on survival and liver function in infants with raidly progressive lysosomal acid lipase deficiency

(2015) S. Jones et al.   JOURNAL OF HEPATOLOGY

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

(2015) Barbara K. Burton et al.   NEW ENGLAND JOURNAL OF MEDICINE

Lysosomal acid lipase deficiency – An under-recognized cause of dyslipidaemia and liver dysfunction

(2014) Željko Reiner et al.   ATHEROSCLEROSIS

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

(2014) Vassili Valayannopoulos et al.   JOURNAL OF HEPATOLOGY

Therapy for lysosomal acid lipase deficiency: Replacing a missing link

(2013) Gregory Grabowski   HEPATOLOGY

Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients With cholesteryl ester storage disease

(2013) Manisha Balwani et al.   HEPATOLOGY

Cholesteryl ester storage disease: Review of the findings in 135 reported patients with an underdiagnosed disease

(2013) Donna L. Bernstein et al.   JOURNAL OF HEPATOLOGY

Hepatic cholesteryl ester accumulation in lysosomal acid lipase deficiency: Non-invasive identification and treatment monitoring by magnetic resonance

(2013) Peter E. Thelwall et al.   JOURNAL OF HEPATOLOGY

900 RECOMBINANT LYSOSOMAL ACID LIPASE NORMALIZES LIVER WEIGHT, TRANSAMINASES AND HISTOPATHOLOGICAL ABNORMALITIES IN AN MODEL OF CHOLESTERYL ESTER STORAGE DISEASE

(2011) M. Leavitt et al.   JOURNAL OF HEPATOLOGY