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Safety of sebelipase alfa for the treatment of lysosomal acid lipase deficiency
Expert Opinion on Drug Safety, 2021Lysosomal acid lipase deficiency is an autosomal recessive progressive lysosomal storage disease that mainly affects the liver, intestine growth, and causes dyslipidemia. The disease presents as two major phenotypes: the severe early-onset and late-onset forms.
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Sebelipase Alfa: A Review in Lysosomal Acid Lipase Deficiency
American Journal of Cardiovascular Drugs, 2016Sebelipase alfa (Kanuma®, Kanuma™), the first commercially available recombinant human lysosomal acid lipase (LAL), is approved in various countries worldwide, including those of the EU, the USA and Japan, as a long-term enzyme replacement therapy for patients diagnosed with LAL deficiency (LAL-D), an ultra-rare, autosomal recessive, progressive ...
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5PSQ-007 Sebelipase alfa as enzyme replacement therapy in three paediatric patients [PDF]
European Journal of Hospital Pharmacy, 2019 Background Lysosomal acid lipase deficiency (LALD) is a rare lysosomal disorder characterised by clinical with dyslipidaemia and steatohepatitis. Sebelipase-alfa is a recombinant human LAL, recently approved for clinical use in LALD. Purpose Evaluation of the effectiveness and safety of sebelipase-alfa as enzyme replacement therapy in paediatric ...
C Jimenez Nunez +7 more
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Sebelipase Alfa Improves Dyslipidemia in Patients with Cholesteryl Ester Storage Disease
Journal of Clinical Lipidology, 2013Radhika Tripuraneni, MD, MPH, Reena Sharma, MD, Manisha Balwani, MD, MS, Chris Bourdon, MD, Simeon Boyd, MD, Catherine Breen, MD, Anthony Quinn, MBChB, PhD, Eugene Schneider, MD, John Kane, MD, PhD, Bruce Kessler, MD, Patrick Deegan, MD, Greg Enns, MD, Eveline Stock, MD, Tomas Honzik, MD, PhD, V era Malinov a, MuDr., Chet Whitley, MD, PhD, Vassili ...
Radhika Tripuraneni +17 more
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Clinics and Research in Hepatology and Gastroenterology, 2018
Two unrelated infants were diagnosed with and initially treated for hemophagocytic lymphohistiocytosis (HLH), but progressed to cholestasis and liver failure. Early onset lysosomal acid lipase deficiency (EO-LAL-D) was suspected due to lymphocytes with cytoplasmic vacuolation and/or adrenal calcifications and confirmed by enzymatic and genetic analysis.
Anna Tylki-Szymanska +2 more
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Two unrelated infants were diagnosed with and initially treated for hemophagocytic lymphohistiocytosis (HLH), but progressed to cholestasis and liver failure. Early onset lysosomal acid lipase deficiency (EO-LAL-D) was suspected due to lymphocytes with cytoplasmic vacuolation and/or adrenal calcifications and confirmed by enzymatic and genetic analysis.
Anna Tylki-Szymanska +2 more
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Sebelipase alfa: Enzymatic replacement treatment for lysosomal acid lipase deficiency
Drugs of Today, 2016Sebelipase alfa was approved for use in 2015 for patients suffering from lysosomal acid lipase deficiency in either of its two forms. The more severe, early-onset form, Wolman disease, occurs in young infants in whom it is normally fatal within the first year of life.
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Molecular Genetics and Metabolism, 2015
CO RR EC TE D P RO OF in February, 2013. Responses were recorded and qualitative analysis (textual analysis) was performed to identify emerging themes. Results: 43 questionnaires were analyzed in April, 2013. Pain, fatigue, burden of the disease, burden of the genetics, quality of life and chronicity were the main representations identified.
Simon A. Jones +11 more
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CO RR EC TE D P RO OF in February, 2013. Responses were recorded and qualitative analysis (textual analysis) was performed to identify emerging themes. Results: 43 questionnaires were analyzed in April, 2013. Pain, fatigue, burden of the disease, burden of the genetics, quality of life and chronicity were the main representations identified.
Simon A. Jones +11 more
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Twice weekly dosing with sebelipase alfa rescues severely ill infants with Wolman disease
Molecular Genetics and Metabolism, 2023Maria Jose De Castro Lopez +6 more
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