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

Author:

Valayannopoulos Vassili,Malinova Vera,Honzík Tomas,Balwani Manisha,Breen Catherine,Deegan Patrick B.,Enns Gregory M.,Jones Simon A.,Kane John P.,Stock Eveline O.,Tripuraneni Radhika,Eckert Stephen,Schneider Eugene,Hamilton Gavin,Middleton Michael S.,Sirlin Claude,Kessler Bruce,Bourdon Christopher,Boyadjiev Simeon A.,Sharma Reena,Twelves Chris,Whitley Chester B.,Quinn Anthony G.

Publisher

Elsevier BV

Subject

Hepatology

Reference29 articles.

1. Tissue and cellular specific expression of murine lysosomal acid lipase mRNA and protein;Du;J Lipid Res,1996

2. Cholesteryl ester storage disease: review of the findings in 135 reported patients with an under-diagnosed disease;Bernstein;J Hepatol,2013

3. Lysosomal acid lipase deficiencies: the Wolman disease/cholesteryl ester storage disease spectrum;Grabowski,2012

4. Enzyme deficiency in cholesteryl ester storage disease;Sloan;J Clin Invest,1972

5. Cholesterol ester storage disease: clinical, biochemical, and pathological studies;Beaudet;J Pediatr,1977

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