| 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 : |
| PubMedSearch : Burton_2015_N.Engl.J.Med_373_1010 |
| PubMedID: 26352813 |
| Gene_locus related to this paper: human-LIPA |
| 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 : |
| PubMedSearch : Burton_2015_N.Engl.J.Med_373_1010 |
| PubMedID: 26352813 |
| Gene_locus related to this paper: human-LIPA |
| 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 : |
| PubMedSearch : Burton_2015_N.Engl.J.Med_373_1010 |
| PubMedID: 26352813 |
| Gene_locus related to this paper: human-LIPA |
| Title : Orthotopic liver transplantation in an adult with cholesterol ester storage disease - Ambler_2013_JIMD.Rep_8_41 |
| Author(s) : Ambler GK , Hoare M , Brais R , Shaw A , Butler A , Flynn P , Deegan P , Griffiths WJ |
| Ref : JIMD Rep , 8 :41 , 2013 |
| Abstract : |
| PubMedSearch : Ambler_2013_JIMD.Rep_8_41 |
| PubMedID: 23430518 |
| Title : Orthotopic liver transplantation in an adult with cholesterol ester storage disease - Ambler_2013_JIMD.Rep_8_41 |
| Author(s) : Ambler GK , Hoare M , Brais R , Shaw A , Butler A , Flynn P , Deegan P , Griffiths WJ |
| Ref : JIMD Rep , 8 :41 , 2013 |
| Abstract : |
| PubMedSearch : Ambler_2013_JIMD.Rep_8_41 |
| PubMedID: 23430518 |
| Title : Orthotopic liver transplantation in an adult with cholesterol ester storage disease - Ambler_2013_JIMD.Rep_8_41 |
| Author(s) : Ambler GK , Hoare M , Brais R , Shaw A , Butler A , Flynn P , Deegan P , Griffiths WJ |
| Ref : JIMD Rep , 8 :41 , 2013 |
| Abstract : |
| PubMedSearch : Ambler_2013_JIMD.Rep_8_41 |
| PubMedID: 23430518 |