Results 71 to 80 of about 353 (110)

Sebelipase alfa in children and adults with lysosomal acid lipase deficiency: Final results of the ARISE study [PDF]

Journal of Hepatology, 2022
Children and adults with lysosomal acid lipase deficiency (LAL-D) experience cirrhosis and dyslipidemia from lysosomal accumulation of cholesteryl esters and triglycerides. Sebelipase alfa enzyme replacement therapy is indicated for individuals with LAL-D.
Manisha Balwani, François Feillet, Katryn N Furuya   +2 more
exaly   +3 more sources

Sebelipase alfa improves atherogenic biomarkers in adults and children with lysosomal acid lipase deficiency [PDF]

Journal of Clinical Lipidology, 2018
Measures of atherogenic cholesterol, with and without concomitant use of lipid-lowering medications (LLMs), are reported with up to 52 weeks of sebelipase alfa treatment in children and adults with lysosomal acid lipase deficiency (LAL-D) participating in the phase 3 Acid Lipase Replacement Investigating Safety and Efficacy study (NCT01757184).To ...
Tyler Hamby, Don P Wilson, Sachin Marulkar   +2 more
exaly   +3 more sources

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

New England Journal of Medicine, 2015
Lysosomal acid lipase is an essential lipid-metabolizing enzyme that breaks down endocytosed lipid particles and regulates lipid metabolism. We conducted a phase 3 trial of enzyme-replacement therapy in children and adults with lysosomal acid lipase deficiency, an underappreciated cause of cirrhosis and severe dyslipidemia.In this multicenter ...
Manisha Balwani, François Feillet, Katryn N Furuya   +2 more
exaly   +8 more sources

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Successful sebelipase alfa desensitization in a pediatric patient

Journal of Allergy and Clinical Immunology: in Practice, 2019
Emine Dibek Mısırlıoğlu, Betül Siyah Bilgin, İlknur Külhaş Çelik   +2 more
exaly   +3 more sources

Sebelipase Alfa: First Global Approval

Drugs, 2015
Sebelipase alfa (Kanuma™) is a recombinant human lysosomal acid lipase (LAL) developed by Synageva BioPharma Corp. (now Alexion Pharmaceuticals, Inc.) for long-term enzyme replacement therapy in patients with LAL deficiency. The agent, administered by intravenous infusion once weekly or once every other week, acts to replace the deficient enzyme ...
Matt Shirley
exaly   +3 more sources