Enzyme replacement therapy for lysosomal storage disorders

Human Gene Therapy

Volumn 19, Issue 8, 2008, Pages 857-

  Keutzer, Joan ^a   Yee, John ^a  

^a GENZYME CORPORATION   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE BETA; LARONIDASE; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE;

DRUG APPROVAL; DRUG EFFICACY; DRUG INDICATION; ENZYME REPLACEMENT; FABRY DISEASE; FOOD AND DRUG ADMINISTRATION; GENE THERAPY; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; LETTER; LYSOSOME STORAGE DISEASE; NEWBORN SCREENING; TREATMENT INDICATION;

ENZYMES; HUMANS; LYSOSOMAL STORAGE DISEASES;

EID: 50649102135     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2008.0714     Document Type: Letter

References (9)

1. Banikazemi, M., Bultas, J., Waldek, S., Wilcox, W.R., Whitley, C.B., McDonald, M., Finkel, R., Packman, S., Bichet, D.G., Warnock, D.G., and Desnick, R.J. (2007). Agalsidase-β therapy for advanced Fabry disease: A randomized trial. Ann. Intern. Med. 146, 77-86.
2. Eng, C.M., Banikazemi, M., Gordon, R.E., Goldman, M., Phelps, R., Kim, L., Gass, A., Winston, J., Dikman, S., Fallon, J.T., Brodie, S., Stacy, C.B., Mehta, D., Parsons, R., Norton, K., O'Callaghan, M., and Desnick, R.J. (2001a). A phase 1/2 clinical trial of enzyme replacement in Fabry disease: Pharmacokinetic, substrate clearance, and safety studies. Am. J. Hum. Genet. 68, 711-722.
3. Eng, C.M., Guffon, N., Wilcox, W.R., Germain, D.P., Lee, P., Waldek, S., Caplan, L., Linthorst, G.E., and Desnick, R.J. (2001b). Safety and efficacy of recombinant human α-galactosidase A: Replacement therapy in Fabry's disease. N. Engl. J. Med. 345, 9-16.
4. Kakkis, E.D., Muenzer, J., Tiller, G.E., Waber, L., Belmont, J., Passage, M., Izykowski, B., Phillips, J., Doroshow, R., Walot, I., Hoft, R., and Neufeld, E.F. (2001). Enzyme-replacement therapy in mucopolysaccharidosis I. N. Engl. J. Med. 344, 182-188.
5. Kishnani, P.S., Nicolino, M., Voit, T., Rogers, R.C., Tsai, A.C., Waterson, J., Herman, G.E., Amalfitano, A., Thurberg, B.L., Richards, S., Davison, M., Corzo, D., and Chen, Y.T. (2006). Chinese hamster ovary cell-derived recombinant human acid α-glucosidase in infantile-onset Pompe disease. J. Pediatr. 149, 89-97.
6. Kishnani, P.S., Corzo, D., Nicolino, M., Byrne, B., Mandel, H., Hwu, W.L., Leslie, N., Levine, J., Spencer, C., McDonald, M., Li, J., Dumontier, J., Halberthal, M., Chien, Y.H., Hopkin, R., Vijayaraghavan, S., Gruskin, D., Bartholomew, D., van der Ploeg, A., Clancy, J.P., Parini, R., Morin, G., Beck, M., de la Gastine, G.S., Jokic, M., Thurberg, B., Richards, S., Bali, D., Davison, M., Worden, M.A., Chen, Y.T., and Wraith, J.E. (2007). Recombinant human acid α-glucosidase: Major clinical benefits in infantile-onset Pompe disease. Neurology 68, 99-109.
7. Wilcox, W.R., Banikazemi, M., Guffon, N., Waldek, S., Lee, P., Linthorst, G.E., Desnick, R.J., and Germain, D.P. (2004). Long-term safety and efficacy of enzyme replacement therapy for Fabry disease. Am. J. Hum. Genet. 75, 65-74.
8. Wraith, J.E., Clarke, L.A., Beck, M., Kolodny, E.H., Pastores, G.M., Muenzer, J., Rapoport, D.M., Berger, K.I., Swiedler, S.J., Kakkis, E.D., Braakman, T., Chadbourne, E., Walton-Bowen, K., and Cox, G.F. (2004). Enzyme replacement therapy for mucopolysaccharidosis I: A randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase). J. Pediatr. 144, 581-588.
9. Wraith, J.E., Beck, M., Lane, R., van der Ploeg, A.T., Shapiro, E., Xue, Y., Kakkis, E., and Guffon, N. (2007). Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: Results of a multinational study of recombinant human α-L-iduronidase (laronidase). Pediatrics 120, e37-e46.