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).
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Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type IEnzyme replacement therapy with laronidase (Aldurazyme) for treating mucopolysaccharidosis type IEnzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type ILess Is More: Substrate Reduction Therapy for Lysosomal Storage DisordersAttenuated osteoarticular phenotype of type VI mucopolysaccharidosis: a report of four patients and a review of the literatureEffects of enzyme replacement therapy started late in a murine model of mucopolysaccharidosis type IA novel GUSB mutation in Brazilian terriers with severe skeletal abnormalities defines the disease as mucopolysaccharidosis VIIImproved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice.Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I.Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits.Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.Open issues in Mucopolysaccharidosis type I-HurlerImmunomodulatory gene therapy in lysosomal storage disordersMethods of neurodevelopmental assessment in children with neurodegenerative disease: Sanfilippo syndrome.A modified liquid chromatography/tandem mass spectrometry method for predominant disaccharide units of urinary glycosaminoglycans in patients with mucopolysaccharidoses.Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure.Mucopolysaccharidoses in northern Brazil: Targeted mutation screening and urinary glycosaminoglycan excretion in patients undergoing enzyme replacement therapy.The natural history of MPS I: global perspectives from the MPS I Registry.Management of mucopolysaccharidosis type IH (Hurler's syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution's experience.Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type IDelivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.An overview of Korean patients with mucopolysaccharidosis and collaboration through the Asia Pacific MPS Network.Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment.The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UKIdentification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders.Mesenchymal stem cells do not prevent antibody responses against human α-L-iduronidase when used to treat mucopolysaccharidosis type I.Long-term functional outcomes of children with hurler syndrome treated with unrelated umbilical cord blood transplantation.Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and managementSleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome.Capturing phenotypic heterogeneity in MPS I: results of an international consensus procedure.Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships.Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy.Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type IIEnzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndromeMucopolysaccharidosis: cardiologic features and effects of enzyme-replacement therapy in 24 children with MPS I, II and VI.Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.Diagnostic Approaches to Pediatric Cardiomyopathy of Metabolic Genetic Etiologies and Their Relation to TherapySuccessful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease.Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants.
P2860
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P2860
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).
description
2007 nî lūn-bûn
@nan
2007年の論文
@ja
2007年学术文章
@wuu
2007年学术文章
@zh
2007年学术文章
@zh-cn
2007年学术文章
@zh-hans
2007年学术文章
@zh-my
2007年学术文章
@zh-sg
2007年學術文章
@yue
2007年學術文章
@zh-hant
name
Enzyme replacement therapy in ...... ha-L-iduronidase (laronidase).
@en
Enzyme replacement therapy in ...... nant human alpha-L-iduronidase
@nl
type
label
Enzyme replacement therapy in ...... ha-L-iduronidase (laronidase).
@en
Enzyme replacement therapy in ...... nant human alpha-L-iduronidase
@nl
prefLabel
Enzyme replacement therapy in ...... ha-L-iduronidase (laronidase).
@en
Enzyme replacement therapy in ...... nant human alpha-L-iduronidase
@nl
P2093
P356
P1433
P1476
Enzyme replacement therapy in ...... ha-L-iduronidase (laronidase).
@en
P2093
Ans van der Ploeg
Elsa Shapiro
Emil D Kakkis
J Edmond Wraith
Michael Beck
Nathalie Guffon
Roderick Lane
P304
P356
10.1542/PEDS.2006-2156
P407
P577
2007-06-04T00:00:00Z