Mukopolisakkaridozlar
Özet
Referanslar
Safary A, Moghaddas-Sani H, Akbarzadeh-Khiavi M, Khabbazzi A, Rafi MA, Omidi Y. Enzyme replacement combinational therapy: effective treatments for mucopolysaccharidoses. Expert Opin Biol Ther. 2021;21(9):1181-1197.
Dietz HC. New therapeutic approaches to mendelian disorders. N Engl J Med. 2010;363(9):852-863.
Kakkis ED, Muenzer J, Tiller GE, et al. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med. 2001;344(3):182-188.
Wraith JE, Clarke LA, Beck M, et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr. 2004;144(5):581-588.
Sifuentes M, Doroshow R, Hoft R, et al. A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years. Mol Genet Metab. 2007;90(2):171-180.
Referanslar
Safary A, Moghaddas-Sani H, Akbarzadeh-Khiavi M, Khabbazzi A, Rafi MA, Omidi Y. Enzyme replacement combinational therapy: effective treatments for mucopolysaccharidoses. Expert Opin Biol Ther. 2021;21(9):1181-1197.
Dietz HC. New therapeutic approaches to mendelian disorders. N Engl J Med. 2010;363(9):852-863.
Kakkis ED, Muenzer J, Tiller GE, et al. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med. 2001;344(3):182-188.
Wraith JE, Clarke LA, Beck M, et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr. 2004;144(5):581-588.
Sifuentes M, Doroshow R, Hoft R, et al. A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years. Mol Genet Metab. 2007;90(2):171-180.
