| Title : Long-term Sebelipase Alfa Treatment in Children and Adults With Lysosomal Acid Lipase Deficiency - Burton_2022_J.Pediatr.Gastroenterol.Nutr__ |
| Author(s) : Burton BK , Sanchez AC , Kostyleva M , Martins AM , Marulkar S , Abel F , Baric I |
| Ref : J Pediatr Gastroenterol Nutr , : , 2022 |
| PubMedID: 35442238 |
| Title : Sebelipase alfa improves atherogenic biomarkers in adults and children with lysosomal acid lipase deficiency - Wilson_2018_J.Clin.Lipidol_12_604 |
| Author(s) : Wilson DP , Friedman M , Marulkar S , Hamby T , Bruckert E |
| Ref : J Clin Lipidol , 12 :604 , 2018 |
| PubMedID: 29628368 |
| Gene_locus related to this paper: human-LIPA |
| Title : Progression of liver disease in children and adults with lysosomal acid lipase deficiency - Burton_2017_Curr.Med.Res.Opin__1 |
| Author(s) : Burton BK , Silliman N , Marulkar S |
| Ref : Curr Med Res Opin , :1 , 2017 |
| PubMedID: 28320214 |
| Title : Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study - Jones_2017_Orphanet.J.Rare.Dis_12_25 |
| Author(s) : Jones SA , Rojas-Caro S , Quinn AG , Friedman M , Marulkar S , Ezgu F , Zaki O , Gargus JJ , Hughes J , Plantaz D , Vara R , Eckert S , Arnoux JB , Brassier A , Le Quan Sang KH , Valayannopoulos V |
| Ref : Orphanet J Rare Dis , 12 :25 , 2017 |
| PubMedID: 28179030 |
| Gene_locus related to this paper: human-LIPA |