Synergy between the pharmacological chaperone 1-deoxygalactonojirimycin and agalsidase alpha in cultured fibroblasts from patients with Fabry disease

Journal of Inherited Metabolic Disease

Volumn 37, Issue 1, 2014, Pages 145-146

  Pisani, Antonio ^a   Porto, Caterina ^a,b   Andria, Generoso ^a   Parenti, Giancarlo ^a,b  

^a UNIVERSITY OF NAPLES FEDERICO II   (Italy)

^b TELETHON INSTITUTE OF GENETICS AND MEDICINE TIGEM   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE ALFA; CHAPERONE; MIGALASTAT; RECOMBINANT ENZYME;

CONTROLLED STUDY; ENZYME ACTIVITY; ENZYME REPLACEMENT; FABRY DISEASE; FIBROBLAST CULTURE; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; HUMAN CELL; LETTER; LYSOSOME STORAGE DISEASE;

1-DEOXYNOJIRIMYCIN; ALPHA-GALACTOSIDASE; FABRY DISEASE; FIBROBLASTS; HUMANS; MALE; RECOMBINANT PROTEINS;

EID: 84891824984     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-013-9641-z     Document Type: Letter

References (2)

1. Porto C, Pisani A, Rosa M et al (2012) Synergy between the pharmacological chaperone 1-deoxygalactonojirimycin and the human recombinant alpha-galactosidase A in cultured fibroblasts from patients with Fabry disease. J Inher Metab Dis 35:513-520
2. Porto C, Cardone M, Fontana F et al (2009) The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts. Mol Ther 17:964-971