Published in Mol Genet Metab Rep on June 08, 2017
Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase). Pediatrics (2007) 2.22
Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study. Blood (2015) 1.78
Mucopolysaccharidosis type II: an update on mutation spectrum. Acta Paediatr Suppl (2007) 1.59
Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant (2003) 1.54
Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure. Orphanet J Rare Dis (2011) 1.54
Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy. J Pediatr (2009) 1.52
Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources. Bone Marrow Transplant (2003) 1.50
Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: a nationwide survey in Japan. Mol Genet Metab (2012) 1.30
The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus. Eur J Pediatr (2011) 1.27
Long-term follow-up following bone marrow transplantation for Hunter disease. J Inherit Metab Dis (1999) 1.27
Pharmacological drugs inducing ototoxicity, vestibular symptoms and tinnitus: a reasoned and updated guide. Eur Rev Med Pharmacol Sci (2011) 1.23
Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17 years. J Pediatr (2009) 1.19
Natural progression of neurological disease in mucopolysaccharidosis type II. Pediatrics (2011) 1.03
Early treatment is associated with improved cognition in Hurler syndrome. Ann Neurol (2014) 0.97
Blood-brain barrier molecular trojan horse enables imaging of brain uptake of radioiodinated recombinant protein in the rhesus monkey. Bioconjug Chem (2013) 0.90
Placenta analysis of prenatally diagnosed patients reveals early GAG storage in mucopolysaccharidoses II and VI. Mol Genet Metab (2011) 0.89
Bone marrow transplantation in Hunter syndrome. J Pediatr (1996) 0.88
Enzyme Replacement Therapy in Mucopolysaccharidosis II Patients Under 1 Year of Age. JIMD Rep (2014) 0.88
Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10year combined experience of 2 centres. Mol Genet Metab (2016) 0.88
Stem cell transplantation in inherited metabolic disorders. Hematology Am Soc Hematol Educ Program (2011) 0.86
Mucopolysaccharidosis type II: identification of 30 novel mutations among Latin American patients. Mol Genet Metab (2013) 0.83
Activities of daily living in patients with Hunter syndrome: impact of enzyme replacement therapy and hematopoietic stem cell transplantation. Mol Genet Metab (2014) 0.83
Otologic and laryngologic manifestations of mucopolysaccharidoses after bone marrow transplantation. Otolaryngol Head Neck Surg (1998) 0.82
Allogeneic Hematopoietic Stem Cell Transplantation in Thirty-Four Pediatric Cases of Mucopolysaccharidosis-A Ten-Year Report from the China Children Transplant Group. Biol Blood Marrow Transplant (2016) 0.82
Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case series. J Inherit Metab Dis (2014) 0.82
Cord blood is the optimal graft source for the treatment of pediatric patients with lysosomal storage diseases: clinical outcomes and future directions. Cytotherapy (2015) 0.81
Clinical, biochemical and molecular characteristics of Filipino patients with mucopolysaccharidosis type II - Hunter syndrome. Orphanet J Rare Dis (2017) 0.78
Improvements in sensorineural hearing loss after cord blood transplant in patients with mucopolysaccharidosis. Arch Otolaryngol Head Neck Surg (2012) 0.77