Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
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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 IEnzyme replacement therapy with laronidase (Aldurazyme ® ) for treating mucopolysaccharidosis type ITherapeutic potential of intracerebroventricular replacement of modified human β-hexosaminidase B for GM2 gangliosidosisHealth-related quality of life in mucopolysaccharidosis: looking beyond biomedical issuesEffects of enzyme replacement therapy started late in a murine model of mucopolysaccharidosis type IDose responsive effects of subcutaneous pentosan polysulfate injection in mucopolysaccharidosis type VI rats and comparison to oral treatmentA novel GUSB mutation in Brazilian terriers with severe skeletal abnormalities defines the disease as mucopolysaccharidosis VIIRespiratory and sleep disorders in mucopolysaccharidosisA self-inactivating gamma-retroviral vector reduces manifestations of mucopolysaccharidosis I in miceSix-minute walk test: reference values and prediction equation in healthy boys aged 5 to 12 years.Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfataseOpen issues in Mucopolysaccharidosis type I-HurlerChildhood onset of Scheie syndrome, the attenuated form of mucopolysaccharidosis I.Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase.Assessment of musculoskeletal abnormalities in children with mucopolysaccharidoses using pGALSA 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.Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry.The natural history of MPS I: global perspectives from the MPS I Registry.Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type IEfficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study.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.Research challenges in central nervous system manifestations of inborn errors of metabolismIdentification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders.Clinical manifestations and treatment of mucopolysaccharidosis type I patients in Latin America as compared with the rest of the world.Glycosaminoglycan storage disorders: a reviewUltrasonographic Features of Hip Joints in Mucopolysaccharidoses Type I and II.Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome.12 year follow up of enzyme-replacement therapy in two siblings with attenuated mucopolysaccharidosis I: the important role of early treatment.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 IICombined Enzyme Replacement Therapy and Hematopoietic Stem Cell Transplantation in Mucopolysacharidosis Type VI.IgG-enzyme fusion protein: pharmacokinetics and anti-drug antibody response in rhesus monkeys.A Dysmorphometric Analysis to Investigate Facial Phenotypic Signatures as a Foundation for Non-invasive Monitoring of Lysosomal Storage Disorders.Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.
P2860
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P2860
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
description
2009 nî lūn-bûn
@nan
2009年の論文
@ja
2009年学术文章
@wuu
2009年学术文章
@zh
2009年学术文章
@zh-cn
2009年学术文章
@zh-hans
2009年学术文章
@zh-my
2009年学术文章
@zh-sg
2009年學術文章
@yue
2009年學術文章
@zh-hant
name
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
@en
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
@nl
type
label
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
@en
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
@nl
prefLabel
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
@en
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
@nl
P2093
P50
P356
P1433
P1476
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
@en
P2093
Edwin H Kolodny
Emil D Kakkis
Gerald F Cox
Gregory M Pastores
J Edmond Wraith
Joseph Muenzer
Marisa Sidman
Michael Beck
P304
P356
10.1542/PEDS.2007-3847
P407
P577
2009-01-01T00:00:00Z