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BONE MARROW TRANSPLANTATION (2007)
A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years
Monica Sifuentes et al.
MOLECULAR GENETICS AND METABOLISM (2007)
Biochemical monitoring after haemopoietic stem cell transplant for Hurler syndrome (MPSIH): implications for functional outcome after transplant in metabolic disease
H. Church et al.
BONE MARROW TRANSPLANTATION (2007)
Ocular findings in four children with mucopolysaccharidosis I-Hurler (MPS I-H) treated early with haematopoietic stem cell transplantation
Kristina Tear Fahnehjelm et al.
ACTA OPHTHALMOLOGICA SCANDINAVICA (2006)
Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I
Elizabeth A. Braunlin et al.
AMERICAN JOURNAL OF CARDIOLOGY (2006)
Long-term outcomes of adaptive functions for children with mucopolysaccharidosis I (Hurler syndrome) treated with hematopoietic stem cell transplantation
Kendra J. Bjoraker et al.
JOURNAL OF DEVELOPMENTAL AND BEHAVIORAL PEDIATRICS (2006)
Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome
J. Cox-Brinkman et al.
BONE MARROW TRANSPLANTATION (2006)
Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII
C Vogler et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)
Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome
SS Grewal et al.
GENETICS IN MEDICINE (2005)
Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice
YL Liu et al.
MOLECULAR THERAPY (2005)
Successful induction of immune tolerance to enzyme replacement therapy in canine mucopolysaccharidosis I
E Kakkis et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2004)
Glycosaminoglycans and proteoglycans in normal mitral valve leaflets and chordae: association with regions of tensile and compressive loading
KJ Grande-Allen et al.
GLYCOBIOLOGY (2004)
Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human α-L-iduronidase gene
SD Hartung et al.
MOLECULAR THERAPY (2004)
Cord-blood transplants from unrelated donors in patients with Hurler's syndrome
SL Staba et al.
NEW ENGLAND JOURNAL OF MEDICINE (2004)
Enzyme replacement therapy for mucopolysaccharidosis I:: A randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (Laronidase)
JE Wraith et al.
JOURNAL OF PEDIATRICS (2004)
Musculoskeletal manifestations of hurler syndrome - Long-term follow-up after bone marrow transplantation
JS Weisstein et al.
JOURNAL OF PEDIATRIC ORTHOPAEDICS (2004)
Usefulness of bone marrow transplantation in the Hurler syndrome
EA Braunlin et al.
AMERICAN JOURNAL OF CARDIOLOGY (2003)
Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources
G Souillet et al.
BONE MARROW TRANSPLANTATION (2003)
Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I
R Kakavanos et al.
LANCET (2003)
Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines
C Peters et al.
BONE MARROW TRANSPLANTATION (2003)
In utero injection of α-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I:: Infection of multiple tissues and neonatal gene expression
L Meertens et al.
HUMAN GENE THERAPY (2002)
Coronary artery patency following long-term successful engraftment 14 years after bone marrow transplantation in the Hurler syndrome
EA Braunlin et al.
AMERICAN JOURNAL OF CARDIOLOGY (2001)
Enzyme-replacement therapy in mucopolysaccharidosis I.
ED Kakkis et al.
NEW ENGLAND JOURNAL OF MEDICINE (2001)
Correction of odontoid dysplasia following bone-marrow transplantation and engraftment (in Hurler syndrome MPS 1H)
SH Hite et al.
PEDIATRIC RADIOLOGY (2000)