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Rojas-Caro S

References (2)

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 :
PubMedSearch : Jones_2017_Orphanet.J.Rare.Dis_12_25
PubMedID: 28179030
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