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Clinical effectiveness of enzyme replacement therapy with galsulfase in mucopolysaccharidosis type VI treatment: Systematic review.
Gomes DF, Gallo LG, Leite BF, Silva RB, da Silva EN. Gomes DF, et al. J Inherit Metab Dis. 2019 Jan;42(1):66-76. doi: 10.1002/jimd.12028. J Inherit Metab Dis. 2019. PMID: 30740728
INTRODUCTION: Mucopolysaccharidosis VI is a rare disease characterized by the arylsulfatase B enzyme deficiency, which is responsible for different clinical manifestations. ...OBJECTIVE: Evaluate the effectiveness of the enzyme replacement therapy with galsulfase fo …
INTRODUCTION: Mucopolysaccharidosis VI is a rare disease characterized by the arylsulfatase B enzyme deficiency, which is resp …
Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI.
Brunelli MJ, Atallah ÁN, da Silva EM. Brunelli MJ, et al. Cochrane Database Syst Rev. 2016 Mar 4;3:CD009806. doi: 10.1002/14651858.CD009806.pub2. Cochrane Database Syst Rev. 2016. PMID: 26943923 Updated. Review.
BACKGROUND: Mucopolysaccharidosis type VI or Maroteaux-Lamy syndrome is a rare genetic disorder caused by the deficiency of arylsulphatase B. ...Clinical manifestation is typically by two or three years of age; however, slowly progressive cases may not present until …
BACKGROUND: Mucopolysaccharidosis type VI or Maroteaux-Lamy syndrome is a rare genetic disorder caused by the deficiency of ar …
Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI.
Brunelli MJ, Atallah ÁN, da Silva EM. Brunelli MJ, et al. Cochrane Database Syst Rev. 2021 Sep 17;9(9):CD009806. doi: 10.1002/14651858.CD009806.pub3. Cochrane Database Syst Rev. 2021. PMID: 34533215 Free PMC article. Review.
BACKGROUND: Mucopolysaccharidosis type VI (MPS VI) or Maroteaux-Lamy syndrome is a rare genetic disorder caused by the deficiency of arylsulphatase B. ...Enzyme replacement therapy (ERT) with galsulfase is considered a new approach for treating MPS VI. …
BACKGROUND: Mucopolysaccharidosis type VI (MPS VI) or Maroteaux-Lamy syndrome is a rare genetic disorder caused by the …
A systematic review of new advances in the management of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): focus on galsulfase.
El Dib RP, Pastores GM. El Dib RP, et al. Biologics. 2009;3:459-68. doi: 10.2147/btt.2009.3580. Epub 2009 Oct 12. Biologics. 2009. PMID: 19851471 Free PMC article.
INTRODUCTION: Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) is an autosomal recessive lysosomal storage disorder, characterized primarily by skeletal dysplasia and joint contracture. ...This review highlights the need for continued research …
INTRODUCTION: Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) is an autosomal recessive lysosomal stora …
Current and potential therapeutic strategies for mucopolysaccharidoses.
Noh H, Lee JI. Noh H, et al. J Clin Pharm Ther. 2014 Jun;39(3):215-24. doi: 10.1111/jcpt.12136. Epub 2014 Feb 25. J Clin Pharm Ther. 2014. PMID: 24612142 Review.
In the early 2000s, enzyme replacement therapy (ERT) was approved by the United States Food and Drug Administration (FDA) for the treatment of MPS I, II and VI. Clinical trials of ERT showed substantial improvements in patients' somatic symptoms; however, no benefit was fo …
In the early 2000s, enzyme replacement therapy (ERT) was approved by the United States Food and Drug Administration (FDA) for the treatment …
Laronidase for treating mucopolysaccharidosis type I.
El Dib RP, Pastores GM. El Dib RP, et al. Genet Mol Res. 2007 Sep 30;6(3):667-74. Genet Mol Res. 2007. PMID: 18050087 Free article. Review.
Enzyme replacement therapies are currently in use or are being tested for at least three different subtypes (I, II and VI). The aim of the present study was to evaluate the effectiveness and safety of laronidase for treating mucopolysaccharidosis type I. ...Laronida …
Enzyme replacement therapies are currently in use or are being tested for at least three different subtypes (I, II and VI). The aim o …
Surgical Management of Thoracolumbar Kyphosis in Patients With Mucopolysaccharidosis: A Systematic Review.
Williams N, Cundy PJ, Eastwod DM. Williams N, et al. Spine (Phila Pa 1976). 2017 Dec 1;42(23):1817-1825. doi: 10.1097/BRS.0000000000002242. Spine (Phila Pa 1976). 2017. PMID: 28538597
Preoperative neurological compromise associated with thoracolumbar kyphosis was reported only in MPS IV and VI, where it was associated with factors other than the degree of kyphosis. ...
Preoperative neurological compromise associated with thoracolumbar kyphosis was reported only in MPS IV and VI, where it was associat …