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| Title : Lysosomal acid lipase deficiency manifestations in children and adults: Baseline data from an international registry - Balwani_2023_Liver.Int__ |
| Author(s) : Balwani M , Balistreri W , D'Antiga L , Evans J , Ros E , Abel F , Wilson DP |
| Ref : Liver Int , : , 2023 |
| Abstract : Balwani_2023_Liver.Int__ |
| ESTHER : Balwani_2023_Liver.Int__ |
| PubMedSearch : Balwani_2023_Liver.Int__ |
| PubMedID: 37222260 |
| Title : A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency - Burton_2015_N.Engl.J.Med_373_1010 |
| Author(s) : Burton BK , Balwani M , Feillet F , Baric I , Burrow TA , Camarena Grande C , Coker M , Consuelo-Sanchez A , Deegan P , Di Rocco M , Enns GM , Erbe R , Ezgu F , Ficicioglu C , Furuya KN , Kane J , Laukaitis C , Mengel E , Neilan EG , Nightingale S , Peters H , Scarpa M , Schwab KO , Smolka V , Valayannopoulos V , Wood M , Goodman Z , Yang Y , Eckert S , Rojas-Caro S , Quinn AG |
| Ref : N Engl J Med , 373 :1010 , 2015 |
| Abstract : Burton_2015_N.Engl.J.Med_373_1010 |
| ESTHER : Burton_2015_N.Engl.J.Med_373_1010 |
| PubMedSearch : Burton_2015_N.Engl.J.Med_373_1010 |
| PubMedID: 26352813 |
| Gene_locus related to this paper: human-LIPA |
| Title : Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency - Valayannopoulos_2014_J.Hepatol_61_1135 |
| Author(s) : Valayannopoulos V , Malinova V , Honzik T , Balwani M , Breen C , Deegan PB , Enns GM , Jones SA , Kane JP , Stock EO , Tripuraneni R , Eckert S , Schneider E , Hamilton G , Middleton MS , Sirlin C , Kessler B , Bourdon C , Boyadjiev SA , Sharma R , Twelves C , Whitley CB , Quinn AG |
| Ref : Journal of Hepatology , 61 :1135 , 2014 |
| Abstract : Valayannopoulos_2014_J.Hepatol_61_1135 |
| ESTHER : Valayannopoulos_2014_J.Hepatol_61_1135 |
| PubMedSearch : Valayannopoulos_2014_J.Hepatol_61_1135 |
| PubMedID: 24993530 |
| Gene_locus related to this paper: human-LIPA |
| Title : Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease - Balwani_2013_Hepatology_58_950 |
| Author(s) : Balwani M , Breen C , Enns GM , Deegan PB , Honzik T , Jones S , Kane JP , Malinova V , Sharma R , Stock EO , Valayannopoulos V , Wraith JE , Burg J , Eckert S , Schneider E , Quinn AG |
| Ref : Hepatology , 58 :950 , 2013 |
| Abstract : Balwani_2013_Hepatology_58_950 |
| ESTHER : Balwani_2013_Hepatology_58_950 |
| PubMedSearch : Balwani_2013_Hepatology_58_950 |
| PubMedID: 23348766 |