| 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 |
| Abstract : Jones_2017_Orphanet.J.Rare.Dis_12_25 |
| ESTHER : Jones_2017_Orphanet.J.Rare.Dis_12_25 |
| PubMedSearch : Jones_2017_Orphanet.J.Rare.Dis_12_25 |
| PubMedID: 28179030 |
| Gene_locus related to this paper: human-LIPA |
| Gene_locus related to this paper: human-LIPA |
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 (2017)
Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study
Orphanet J Rare Dis
12 :25
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 (2017)
Orphanet J Rare Dis
12 :25