Progression of organ manifestations upon enzyme replacement therapy in a patient with mucopolysaccharidosis type I/Hurler

[1] Peters C.(1996)Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome Blood 87 4894-4902

[2] Balthazor M.(2004)Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase) J Pediatr 144 581-588

[3] Shapiro E.G.(2005)Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome Genet Med 7 143-146

[4] King R.J.(1992)alpha-L-iduronidase mutations (Q70X and P533R) associate with a severe Hurler phenotype Hum Mutat 1 333-339

[5] Kollman C.(2007)A follow-up study of MPSI patients treated with laronidase enzyme replacement therapy for 6 years Mol Genet Metab 90 171-180

[6] Hegland J.D.(2006)Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I Am J Cardiol 98 416-418

[7] Wraith J.E.(2007)Enzyme replacement therapy in two patients with an advanced severe (Hurler) phenotype of mucopolysaccharidosis I Eur J Pediatr 166 727-732

[8] Clarke L.A.(2006)Outcome after three years of laronidase enzyme replacement therapy in a patient with Hurler syndrome J Inherit Metab Dis 29 762-188

[9] Beck M.(2001)Enzyme-replacement therapy in mucopolysaccharidosis I N Engl J Med 344 182-undefined

[10] Kolodny E.H.(undefined)undefined undefined undefined undefined-undefined

[1] Peters C.(1996)Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome Blood 87 4894-4902

[2] Balthazor M.(2004)Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase) J Pediatr 144 581-588

[3] Shapiro E.G.(2005)Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome Genet Med 7 143-146

[4] King R.J.(1992)alpha-L-iduronidase mutations (Q70X and P533R) associate with a severe Hurler phenotype Hum Mutat 1 333-339

[5] Kollman C.(2007)A follow-up study of MPSI patients treated with laronidase enzyme replacement therapy for 6 years Mol Genet Metab 90 171-180

[6] Hegland J.D.(2006)Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I Am J Cardiol 98 416-418

[7] Wraith J.E.(2007)Enzyme replacement therapy in two patients with an advanced severe (Hurler) phenotype of mucopolysaccharidosis I Eur J Pediatr 166 727-732

[8] Clarke L.A.(2006)Outcome after three years of laronidase enzyme replacement therapy in a patient with Hurler syndrome J Inherit Metab Dis 29 762-188

[9] Beck M.(2001)Enzyme-replacement therapy in mucopolysaccharidosis I N Engl J Med 344 182-undefined

[10] Kolodny E.H.(undefined)undefined undefined undefined undefined-undefined