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Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita

Bone marrow transplantation (Basingstoke), 2011-01, Vol.46 (1), p.98-104

2011

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Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome

Bone marrow transplantation (Basingstoke), 2008-03, Vol.41 (6), p.531-535

2008

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Changes in the incidence, patterns and outcomes of graft failure following hematopoietic stem cell transplantation for Hurler syndrome

Bone marrow transplantation (Basingstoke), 2017-06, Vol.52 (6), p.846-853

2017

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Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients

Bone marrow transplantation (Basingstoke), 2012-03, Vol.47 (3), p.352-359

2012

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Long-term metabolic, endocrine, and neuropsychological outcome of hematopoietic cell transplantation for Wolman disease

Bone marrow transplantation (Basingstoke), 2009-01, Vol.43 (1), p.21-27

2009

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Defects in TCIRG1 subunit of the vacuolar proton pump are responsible for a subset of human autosomal recessive osteopetrosis

Nature genetics, 2000-07, Vol.25 (3), p.343-346

2000

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Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation

Bone marrow transplantation (Basingstoke), 2008-06, Vol.41 (12), p.1005-1011

2008

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Characterization and management of hypercalcemia following transplantation for osteopetrosis

Bone marrow transplantation (Basingstoke), 2010-05, Vol.45 (5), p.939-944

2010

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Pre-transplant risk factors affecting outcome in Hurler syndrome

Bone marrow transplantation (Basingstoke), 2010-07, Vol.45 (7), p.1239-1246

2010

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No evidence for the reversal of adrenal failure after hematopoietic cell transplantation in X-linked adrenoleukodystrophy

Bone marrow transplantation (Basingstoke), 2012-10, Vol.47 (10), p.1377-1378

2012

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N-acetyl-L-cysteine improves outcome of advanced cerebral adrenoleukodystrophy

Bone marrow transplantation (Basingstoke), 2007-02, Vol.39 (4), p.211-215

2007

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Analysis of factors affecting development of carpal tunnel syndrome in patients with Hurler syndrome after hematopoietic cell transplantation

Bone marrow transplantation (Basingstoke), 2007-03, Vol.39 (6), p.331-334

2007

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Haploidentical transplantation with post-transplant cyclophosphamide following reduced-intensity conditioning for osteopetrosis: outcomes in three children

Bone marrow transplantation (Basingstoke), 2016-11, Vol.51 (11), p.1546-1548

2016

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Intensity of MRI Gadolinium Enhancement in Cerebral Adrenoleukodystrophy: A Biomarker for Inflammation and Predictor of Outcome following Transplantation in Higher Risk Patients

American journal of neuroradiology : AJNR, 2016-02, Vol.37 (2), p.367-372

2016

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Lower leukemia relapse in pediatric patients with pulmonary cytolytic thrombi following allogeneic transplant

Bone marrow transplantation (Basingstoke), 2011-03, Vol.46 (3), p.368-371

2011

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Unrelated donor bone marrow transplantation for children with acute leukemia

Journal of clinical oncology, 1997-02, Vol.15 (2), p.557-565

1997

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Non-myeloablative conditioning for second hematopoietic cell transplantation for graft failure in patients with non-malignant disorders: a prospective study and review of the literature

Bone marrow transplantation (Basingstoke), 2017-05, Vol.52 (5), p.726-732

2017

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Childhood cerebral X-linked adrenoleukodystrophy: diffusion tensor imaging measurements for prediction of clinical outcome after hematopoietic stem cell transplantation

American journal of neuroradiology : AJNR, 2013-03, Vol.34 (3), p.641-649

2013

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Clinical responses to bone marrow transplantation in children with severe osteogenesis imperfecta

Blood, 2001-03, Vol.97 (5), p.1227-1231

2001

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Outcome of second hematopoietic cell transplantation in Hurler syndrome

Bone marrow transplantation (Basingstoke), 2002-03, Vol.29 (6), p.491-496

2002

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Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell Transplantation

JIMD Reports, Volume 29, 2016-01, Vol.29, p.95-102

2016

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Graft-versus-leukemia is sufficient to induce remission in juvenile myelomonocytic leukemia

Bone marrow transplantation (Basingstoke), 1998-07, Vol.22 (2), p.201-203

1998

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Haemopoietic cell transplantation in children with juvenile myelomonocytic leukaemia

British journal of haematology, 1998-11, Vol.103 (2), p.552-558

1998

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Unrelated donor bone marrow transplantation for children and adolescents with aplastic anaemia or myelodysplasia

British journal of haematology, 1997-03, Vol.96 (4), p.749-756

1997

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Hematopoietic stem cell transplantation for infantile osteopetrosis

Bone marrow transplantation (Basingstoke), 1998-11, Vol.22 (10), p.941-946

1998

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Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita

Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome

Changes in the incidence, patterns and outcomes of graft failure following hematopoietic stem cell transplantation for Hurler syndrome

Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients

Long-term metabolic, endocrine, and neuropsychological outcome of hematopoietic cell transplantation for Wolman disease

Defects in TCIRG1 subunit of the vacuolar proton pump are responsible for a subset of human autosomal recessive osteopetrosis

Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation

Characterization and management of hypercalcemia following transplantation for osteopetrosis

Pre-transplant risk factors affecting outcome in Hurler syndrome

No evidence for the reversal of adrenal failure after hematopoietic cell transplantation in X-linked adrenoleukodystrophy

N-acetyl-L-cysteine improves outcome of advanced cerebral adrenoleukodystrophy

Analysis of factors affecting development of carpal tunnel syndrome in patients with Hurler syndrome after hematopoietic cell transplantation

Haploidentical transplantation with post-transplant cyclophosphamide following reduced-intensity conditioning for osteopetrosis: outcomes in three children

Intensity of MRI Gadolinium Enhancement in Cerebral Adrenoleukodystrophy: A Biomarker for Inflammation and Predictor of Outcome following Transplantation in Higher Risk Patients

Lower leukemia relapse in pediatric patients with pulmonary cytolytic thrombi following allogeneic transplant

Unrelated donor bone marrow transplantation for children with acute leukemia

Non-myeloablative conditioning for second hematopoietic cell transplantation for graft failure in patients with non-malignant disorders: a prospective study and review of the literature

Childhood cerebral X-linked adrenoleukodystrophy: diffusion tensor imaging measurements for prediction of clinical outcome after hematopoietic stem cell transplantation

Clinical responses to bone marrow transplantation in children with severe osteogenesis imperfecta

Outcome of second hematopoietic cell transplantation in Hurler syndrome

Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell Transplantation

Graft-versus-leukemia is sufficient to induce remission in juvenile myelomonocytic leukemia

Haemopoietic cell transplantation in children with juvenile myelomonocytic leukaemia

Unrelated donor bone marrow transplantation for children and adolescents with aplastic anaemia or myelodysplasia

Hematopoietic stem cell transplantation for infantile osteopetrosis

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