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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 |