Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).
about
A plant-derived recombinant human glucocerebrosidase enzyme--a preclinical and phase I investigationEnzyme replacement therapy with galsulfase for mucopolysaccharidosis type VIHealth-related quality of life in mucopolysaccharidosis: looking beyond biomedical issuesTherapies for the bone in mucopolysaccharidosesDrug treatment of inborn errors of metabolism: a systematic reviewMorquio A syndrome: diagnosis and current and future therapiesNewborn screening and diagnosis of mucopolysaccharidosesArylsulfatase B improves locomotor function after mouse spinal cord injuryComparative analysis of acid sphingomyelinase distribution in the CNS of rats and mice following intracerebroventricular deliveryA rapid and sensitive method for measuring N-acetylglucosaminidase activity in cultured cellsRespiratory and sleep disorders in mucopolysaccharidosisChinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease.Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transferSimilar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage diseaseThrombocytopenia associated with galsulfase treatment.A selective screening program for the early detection of mucopolysaccharidosis: Results of the FIND project - a 2-year follow-up study.Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfataseMucopolysaccharidosis VI.Bone density assessment in patients with mucopolysaccharidosis: A preliminary report from patients with MPS II and VI.Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase.Hypoxia reduces arylsulfatase B activity and silencing arylsulfatase B replicates and mediates the effects of hypoxia.Tandem mass spectrometry for the direct assay of lysosomal enzymes in dried blood spots: application to screening newborns for mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).Arylsulfatase B regulates versican expression by galectin-3 and AP-1 mediated transcriptional effects.Therapies of mucopolysaccharidosis IVA (Morquio A syndrome).Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016).Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment.Enzyme replacement therapy in newborn mucopolysaccharidosis IVA mice: early treatment rescues bone lesions?Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA.Growth Charts for Individuals with Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome)Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial.Burden of illness of Pompe disease in patients only receiving supportive care.Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome.Pressure for drug development in lysosomal storage disorders - a quantitative analysis thirty years beyond the US orphan drug actCardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and managementDiagnosis of lysosomal storage disorders: current techniques and future directions.Decline in arylsulfatase B and Increase in chondroitin 4-sulfotransferase combine to increase chondroitin 4-sulfate in traumatic brain injury.Production of recombinant beta-hexosaminidase A, a potential enzyme for replacement therapy for Tay-Sachs and Sandhoff diseases, in the methylotrophic yeast Ogataea minutaAnesthesia in a child with Maroteaux-Lamy syndrome undergoing mitral valve replacementMutations in ARSB in MPS VI patients in India.Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8.
P2860
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P2860
Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).
description
2004 nî lūn-bûn
@nan
2004年の論文
@ja
2004年学术文章
@wuu
2004年学术文章
@zh-cn
2004年学术文章
@zh-hans
2004年学术文章
@zh-my
2004年学术文章
@zh-sg
2004年學術文章
@yue
2004年學術文章
@zh
2004年學術文章
@zh-hant
name
Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).
@en
Enzyme replacement therapy in mucopolysaccharidosis VI
@nl
type
label
Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).
@en
Enzyme replacement therapy in mucopolysaccharidosis VI
@nl
prefLabel
Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).
@en
Enzyme replacement therapy in mucopolysaccharidosis VI
@nl
P2093
P1476
Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).
@en
P2093
Barbara Plecko
Chester B Whitley
Ellen Butensky
John J Hopwood
Julie Simon
Lewis Waber
Paige Kaplan
Paul Harmatz
P304
P356
10.1016/J.JPEDS.2004.03.018
P407
P577
2004-05-01T00:00:00Z