Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase.
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Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VITherapies for the bone in mucopolysaccharidosesMorquio A syndrome: diagnosis and current and future therapiesNewborn screening and diagnosis of mucopolysaccharidosesRespiratory and sleep disorders in mucopolysaccharidosisLong-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transferNeonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats.The effect of neonatal gene therapy on skeletal manifestations in mucopolysaccharidosis VII dogs after a decade.The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.Sensory-motor behavioral characterization of an animal model of Maroteaux-Lamy syndrome (or Mucopolysaccharidosis VI).Similar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage diseaseEnzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfataseSix-Minute Walk Test demonstrates motor fatigue in spinal muscular atrophyMucopolysaccharidosis VI.Growth patterns and the use of growth hormone in the mucopolysaccharidoses.Impact of Enzyme Replacement Therapy and Hematopoietic Stem Cell Therapy on Growth in Patients with Hunter Syndrome.Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase.Mucopolysaccharidoses in northern Brazil: Targeted mutation screening and urinary glycosaminoglycan excretion in patients undergoing enzyme replacement therapy.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 (N-acetylgalactosamine-4-sulfatase): potential role as a biomarker in prostate cancer.Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study.Therapies of mucopolysaccharidosis IVA (Morquio A syndrome).Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment.Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseasesIllness perception and clinical treatment experiences in patients with M. Maroteaux-Lamy (mucopolysaccharidosis type VI) and a Turkish migration background in Germany.Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA.Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders.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.The effect of Tlr4 and/or C3 deficiency and of neonatal gene therapy on skeletal disease in mucopolysaccharidosis VII mice.Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome.Glycosaminoglycan storage disorders: a reviewCardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and managementAlpha-mannosidosis: correlation between phenotype, genotype and mutant MAN2B1 subcellular localisation.Decline in arylsulfatase B and Increase in chondroitin 4-sulfotransferase combine to increase chondroitin 4-sulfate in traumatic brain injury.Gene therapy for lysosomal storage diseases (LSDs) in large animal models.Diagnostic and treatment strategies in mucopolysaccharidosis VI.Chronic enzyme replacement therapy ameliorates neuropathology in alpha-mannosidosis miceCombined Enzyme Replacement Therapy and Hematopoietic Stem Cell Transplantation in Mucopolysacharidosis Type VI.Long-term cognitive follow-up in children treated for Maroteaux-Lamy syndrome.
P2860
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P2860
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase.
description
2008 nî lūn-bûn
@nan
2008年の論文
@ja
2008年学术文章
@wuu
2008年学术文章
@zh
2008年学术文章
@zh-cn
2008年学术文章
@zh-hans
2008年学术文章
@zh-my
2008年学术文章
@zh-sg
2008年學術文章
@yue
2008年學術文章
@zh-hant
name
Long-term follow-up of enduran ...... etylgalactosamine 4-sulfatase.
@en
Long-term follow-up of enduran ...... etylgalactosamine 4-sulfatase.
@nl
type
label
Long-term follow-up of enduran ...... etylgalactosamine 4-sulfatase.
@en
Long-term follow-up of enduran ...... etylgalactosamine 4-sulfatase.
@nl
prefLabel
Long-term follow-up of enduran ...... etylgalactosamine 4-sulfatase.
@en
Long-term follow-up of enduran ...... etylgalactosamine 4-sulfatase.
@nl
P2093
P1476
Long-term follow-up of enduran ...... etylgalactosamine 4-sulfatase.
@en
P2093
Barbara Plecko
Celeste Decker
Chester B Whitley
David Ketteridge
Elisa Leão Teles
Ida Vanessa D Schwartz
J Edmond Wraith
John J Hopwood
Laila Arash
M Clara Sá Miranda
P304
P356
10.1016/J.YMGME.2008.04.001
P577
2008-05-23T00:00:00Z