Published in J Inherit Metab Dis on June 09, 2006
Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management. J Inherit Metab Dis (2011) 1.70
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
Lysosomal storage disorders in the newborn. Pediatrics (2009) 1.29
Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment. Genet Mol Biol (2010) 1.07
Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI. Mol Genet Metab (2010) 0.91
Therapies for the bone in mucopolysaccharidoses. Mol Genet Metab (2014) 0.89
Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome. Drug Des Devel Ther (2015) 0.89
Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy. Hum Mol Genet (2015) 0.87
Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome. J Pediatr (2012) 0.87
Prevalence and development of orthopaedic symptoms in the dutch hurler patient population after haematopoietic stem cell transplantation. JIMD Rep (2012) 0.86
Outcome of invasive mechanical ventilation after pediatric allogeneic hematopoietic SCT: results from a prospective, multicenter registry. Bone Marrow Transplant (2014) 0.84
Clinical aspects of neuropathic lysosomal storage disorders. J Inherit Metab Dis (2010) 0.81
Haematopoietic stem cell transplantation does not retard disease progression in the psycho-cognitive variant of late-onset metachromatic leukodystrophy. J Inherit Metab Dis (2010) 0.76
Hematopoietic SCT in Iranian children 1991-2012. Bone Marrow Transplant (2015) 0.75
Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem. Pediatr Endocrinol Rev (2016) 0.75
Implication of cord blood for cell-based therapy in refractory childhood diseases. Int J Stem Cells (2010) 0.75
Clinical utilization of cord blood over human health: experience of stem cell transplantation and cell therapy using cord blood in Korea. Korean J Pediatr (2014) 0.75
The spectrum of leukodystrophies in children: Experience at a tertiary care centre from North India. Ann Indian Acad Neurol (2016) 0.75
Treatment of severe acute graft-versus-host disease with third party haploidentical mesenchymal stem cells. Lancet (2004) 11.77
Transplants of umbilical-cord blood or bone marrow from unrelated donors in adults with acute leukemia. N Engl J Med (2004) 7.73
Outcomes after transplantation of cord blood or bone marrow from unrelated donors in adults with leukemia. N Engl J Med (2004) 7.10
Processing and cryopreservation of placental/umbilical cord blood for unrelated bone marrow reconstitution. Proc Natl Acad Sci U S A (1995) 5.15
Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med (2005) 4.26
Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med (2004) 4.15
Allogeneic mesenchymal stem cell infusion for treatment of metachromatic leukodystrophy (MLD) and Hurler syndrome (MPS-IH). Bone Marrow Transplant (2002) 3.24
Factors associated with outcomes of unrelated cord blood transplant: guidelines for donor choice. Exp Hematol (2004) 2.81
Adrenoleukodystrophy: phenotype, genetics, pathogenesis and therapy. Brain (1997) 2.25
Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation. Lancet (1981) 2.22
Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood (1996) 2.19
Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy. Lancet (2000) 1.98
Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999. Blood (2004) 1.79
Stored placental blood for unrelated bone marrow reconstitution. Blood (1993) 1.72
Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation. J Inherit Metab Dis (1995) 1.62
The measurement of performance in childhood cancer patients. Cancer (1987) 1.56
New trends in umbilical cord blood transplantation. Blood (2005) 1.56
Bone marrow-derived mesenchymal stem cells remain host-derived despite successful hematopoietic engraftment after allogeneic transplantation in patients with lysosomal and peroxisomal storage diseases. Exp Hematol (1999) 1.55
Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant (2003) 1.54
The defect in Hurler and Hunter syndromes. II. Deficiency of specific factors involved in mucopolysaccharide degradation. Proc Natl Acad Sci U S A (1969) 1.53
Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources. Bone Marrow Transplant (2003) 1.50
Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy. N Engl J Med (1998) 1.47
Adrenoleukodystrophy: a scoring method for brain MR observations. AJNR Am J Neuroradiol (1994) 1.47
Bone-marrow transplantation in the Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI). Biochemical and clinical status 24 months after transplantation. N Engl J Med (1984) 1.43
Microglia: the effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases. Cell Transplant (1995) 1.42
Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation. N Engl J Med (1990) 1.41
Induced degradation of glycosaminoglycans in Hurler's and Hunter's syndromes by plasma infusion. Proc Natl Acad Sci U S A (1971) 1.32
Stem cell transplantation (cord blood transplants). Hematology Am Soc Hematol Educ Program (2004) 1.28
High-resolution HLA typing by sequencing for HLA-A, -B, -C, -DR, -DQ in 122 unrelated cord blood/patient pair transplants hardly improves long-term clinical outcome. Bone Marrow Transplant (2005) 1.19
Effect of leukocyte transfusion in a child with type II mucopolysaccharidosis. Proc Natl Acad Sci U S A (1971) 1.06
Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation. Bone Marrow Transplant (1998) 1.02
Hypertrophic cardiomyopathy in mucopolysaccharidoses: regression after bone marrow transplantation. Pediatr Cardiol (1992) 0.98
Coronary artery patency following long-term successful engraftment 14 years after bone marrow transplantation in the Hurler syndrome. Am J Cardiol (2001) 0.93
Ocular changes in the mucopolysaccharidoses after bone marrow transplantation. A preliminary report. Ophthalmology (1989) 0.89
Clinical outcome in four children with metachromatic leukodystrophy treated by bone marrow transplantation. Bone Marrow Transplant (1996) 0.89
Proton MR spectroscopic imaging predicts lesion progression on MRI in X-linked adrenoleukodystrophy. Neurology (2002) 0.88
Hematopoietic cell transplantation activity in Europe for inherited metabolic diseases: open issues and future directions. Bone Marrow Transplant (2005) 0.87
Bone marrow transplantation for Sanfilippo disease type B. J Inherit Metab Dis (1992) 0.84
Childhood cerebral form of adrenoleukodystrophy: short-term effect of bone marrow transplantation on brain MR observations. AJNR Am J Neuroradiol (1994) 0.83
Bone marrow transplantation for the treatment of alpha-mannosidosis. J Pediatr (1998) 0.83
Hurler syndrome: past, present, and future. J Pediatr (1998) 0.83