Ficicioglu C

Title : Lessons Learned from the Long-Term Use of Enzyme Replacement Therapy in the Treatment of Lysosomal Acid Lipase Deficiency -

Author(s) : Strong A , Ficicioglu C

Ref : J Pediatr Gastroenterol Nutr , : , 2022

PubMedID: 35576532

Title : Early diagnosis of infantile-onset lysosomal acid lipase deficiency in the advent of available enzyme replacement therapy - Cohen_2019_Orphanet.J.Rare.Dis_14_198

Author(s) : Cohen JL , Burfield J , Valdez-Gonzalez K , Samuels A , Stefanatos AK , Yudkoff M , Pedro H , Ficicioglu C

Ref : Orphanet J Rare Dis , 14 :198 , 2019

Abstract :

PubMedSearch : Cohen_2019_Orphanet.J.Rare.Dis_14_198

PubMedID: 31412917

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

Ficicioglu C

References (3)

1. Lessons Learned from the Long-Term Use of Enzyme Replacement Therapy in the Treatment of Lysosomal Acid Lipase Deficiency -

2. Early diagnosis of infantile-onset lysosomal acid lipase deficiency in the advent of available enzyme replacement therapy - Cohen_2019_Orphanet.J.Rare.Dis_14_198

3. A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency - Burton_2015_N.Engl.J.Med_373_1010