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

2015 ◽  
Vol 62 ◽  
pp. S811 ◽  
Author(s):  
S. Jones ◽  
D. Plantaz ◽  
R. Vara ◽  
J.J. Gargus ◽  
J. Hughes ◽  
...  
Keyword(s):  
Liver Function ◽  
Acid Lipase ◽  
Lysosomal Acid ◽  
Lysosomal Acid Lipase ◽  
Lysosomal Acid Lipase Deficiency

Effect of sebelipase alfa on survival and liver function in infants with rapidly progressive lysosomal acid lipase deficiency

2015 ◽  
Vol 114 (2) ◽  
pp. S59
Author(s):  
Simon A. Jones ◽  
Dominique Plantaz ◽  
Roshni Vara ◽  
Stephen Eckert ◽  
Kim-Hanh Le Quan Sang ◽  
...  
Keyword(s):  
Liver Function ◽  
Acid Lipase ◽  
Lysosomal Acid ◽  
Lysosomal Acid Lipase ◽  
Lysosomal Acid Lipase Deficiency

Effect of sebelipase alfa on survival to 3 years of age and liver function in infants with rapidly progressive lysosomal acid lipase deficiency

2017 ◽  
Vol 120 (1-2) ◽  
pp. S48
Author(s):  
Mark Friedman ◽  
Simon A. Jones ◽  
Anais Brassier ◽  
Joanne Hughes ◽  
Dominique Plantaz ◽  
...  
Keyword(s):  
Liver Function ◽  
Acid Lipase ◽  
Lysosomal Acid ◽  
Lysosomal Acid Lipase ◽  
Lysosomal Acid Lipase Deficiency

scholarly journals Correction to: Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Suresh Vijay ◽  
Anais Brassier ◽  
Arunabha Ghosh ◽  
Simona Fecarotta ◽  
Florian Abel ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Acid Lipase ◽  
Open Label ◽  
Term Survival ◽  
Lysosomal Acid ◽  
Enzyme Replacement ◽  
Lysosomal Acid Lipase ◽  
Lysosomal Acid Lipase Deficiency

An amendment to this paper has been published and can be accessed via the original article.

scholarly journals The Frequency of Lysosomal Acid Lipase Deficiency in Children With Unexplained Liver Disease

2019 ◽  
Vol 68 (3) ◽  
pp. 371-376
Author(s):  
Zarife Kuloglu ◽  
Aydan Kansu ◽  
Suna Selbuz ◽  
Ayhan G. Kalaycı ◽  
Gülseren Şahin ◽  
...  
Keyword(s):  
Liver Disease ◽  
Acid Lipase ◽  
Lysosomal Acid ◽  
Lysosomal Acid Lipase ◽  
Lysosomal Acid Lipase Deficiency
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