The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I.
about
Attenuation of nonsense-mediated mRNA decay enhances in vivo nonsense suppressionCorneal hysteresis in mucopolysaccharidosis I and VI.Tandem mass spectrometry for the direct assay of lysosomal enzymes in dried blood spots: application to screening newborns for mucopolysaccharidosis IChildhood onset of Scheie syndrome, the attenuated form of mucopolysaccharidosis I.A modified liquid chromatography/tandem mass spectrometry method for predominant disaccharide units of urinary glycosaminoglycans in patients with mucopolysaccharidoses.Management of mucopolysaccharidosis type IH (Hurler's syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution's experience.Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning.Cardiac manifestations in the mouse model of mucopolysaccharidosis I.Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment.Heparan sulfate inhibits hematopoietic stem and progenitor cell migration and engraftment in mucopolysaccharidosis I.Biodistribution and pharmacodynamics of recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrationsClinical manifestations and treatment of mucopolysaccharidosis type I patients in Latin America as compared with the rest of the world.Suppression of nonsense mutations as a therapeutic approach to treat genetic diseases.Suppression of premature termination codons as a therapeutic approach.Two-year follow-up of Sanfilippo Disease patients treated with a genistein-rich isoflavone extract: assessment of effects on cognitive functions and general status of patientsAnimal models for mucopolysaccharidosis disorders and their clinical relevance.The diagnostic journey of patients with mucopolysaccharidosis I: A real-world survey of patient and physician experiences.A pilot newborn screening program for Mucopolysaccharidosis type I in Taiwan.Nonsteroidal anti-inflammatory drugs modulate cellular glycosaminoglycan synthesis by affecting EGFR and PI3K signaling pathwaysNonsense Suppression as an Approach to Treat Lysosomal Storage Diseases.Genetics and metabolic cardiomyopathies.Mucopolysaccharidoses.Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series.Transplantation in inborn errors of metabolism: current considerations and future perspectives.Maternal mosaicism for IDUA deletion clarifies recurrence risk in MPS I.Clinical manifestation of Hurler syndrome in a 7 year old child.Awareness of Fabry disease among rheumatologists--current status and perspectives.Scheie syndrome: enzyme replacement therapy does not prevent progression of cervical myelopathy due to spinal cord compression.Surgical management of thoracolumbar kyphosis in mucopolysaccharidosis type 1 in a reference center.Heparan sulfate derived disaccharides in plasma and total urinary excretion of glycosaminoglycans correlate with disease severity in Sanfilippo disease.Diagnosing lysosomal storage disorders: mucopolysaccharidosis type I.Hematopoietic stem cell transplantation effects on spinal cord compression in Hurler.Idursulfase: enzyme replacement therapy for mucopolysaccharidosis Type II (Hunter syndrome)Substrate accumulation and extracellular matrix remodelling promote persistent upper airway disease in mucopolysaccharidosis patients on enzyme replacement therapy
P2860
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P2860
The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I.
description
2005 nî lūn-bûn
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2005年の論文
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2005年論文
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2005年論文
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2005年論文
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2005年論文
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2005年論文
@zh-tw
2005年论文
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2005年论文
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2005年论文
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name
The first 5 years of clinical ...... y for mucopolysaccharidosis I.
@ast
The first 5 years of clinical ...... y for mucopolysaccharidosis I.
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type
label
The first 5 years of clinical ...... y for mucopolysaccharidosis I.
@ast
The first 5 years of clinical ...... y for mucopolysaccharidosis I.
@en
prefLabel
The first 5 years of clinical ...... y for mucopolysaccharidosis I.
@ast
The first 5 years of clinical ...... y for mucopolysaccharidosis I.
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P2860
P356
P1476
The first 5 years of clinical ...... y for mucopolysaccharidosis I.
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P2093
James Edward Wraith
P2860
P304
P356
10.1517/14656566.6.3.489
P407
P577
2005-03-01T00:00:00Z