70
Views
1
CrossRef citations to date
0
Altmetric
Review

A systematic review of new advances in the management of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): focus on galsulfase

&
Pages 459-468 | Published online: 07 Dec 2022

References

  • HigginsJPTAltmanDGHigginsJPTGreenSChapter 8: Assessing risk of bias in included studiesCochrane Handbook for Systematic Reviews of Interventions Version 5.0.0 (updated February 2008)The Cochrane Collaboration2008 Available from www.cochrane-handbook.org
  • HigginsJPThompsonSGDeeksJJAltmanDGMeasuring inconsistency in meta-analysisBMJ2003327741455756012958120
  • HigginsJPTGreenSAssessment of study qualityCochrane Reviewers’ Handbook 425 The Cochrane Library32005Chichester, UKJohn Wiley & Sons, Ltd2005
  • ScarpaMBaroneRFiumaraAAstaritaLParentiGRampazzoAMucopolysaccharidosis VI: the Italian experienceEur J Pediatr2009 [Epub ahead of print]
  • BagewadiSRobertsJMercerJJonesSStephensonJWraithJEHome treatment with Elaprase and Naglazyme is safe in patients with mucopolysaccharidoses types II and VI, respectivelyJ Inherit Metab Dis200831673373718923918
  • Cardoso-SantosAAzevedoACFagondesSBurinMGGiuglianiRSchwartzIVMucopolysaccharidosis type VI (Maroteaux-Lamy syndrome): assessment of joint mobility and grip and pinch strengthJ Pediatr2008842130135
  • HarmatzPGiuglianiRSchwartzIVGuffonNTelesELMirandaMCLong-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-sulfataseMol Genet Metab200894446947518502162
  • HarmatzPKetteridgeDGiuglianiRGuffonNTelesELMirandaMCDirect comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatasePediatr20051156e681e689
  • AzevedoACSchwartzIVKalakunLBrustolinSBurinMGBeheregarayAPClinical and biochemical study of 28 patients with mucopolysaccharidosis type VIClin Genet200466320821315324318
  • HarmatzPGiuglianiRSchwartzIGuffonNTelesELMirandaMCEnzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension studyJ Pediatr2006148453353916647419
  • HarmatzPWhitleyCBWaberLPaisRSteinerRPleckoBEnzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)J Pediatr2004144557458015126989
  • HarmatzPKramerWGHopwoodJJSimonJButenskyESwiedlerSJPharmacokinetic profile of recombinant human N-acetylgalactosamine 4-sulphatase enzyme replacement therapy in patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): a phase I/II studyActa Paediatr Suppl200594447616815895715
  • Jakóbkiewicz-BaneckaJPiotrowskaENarajczykMBarańskaSWegrzynGGenistein-mediated inhibition of glycosaminoglycan synthesis, which corrects storage in cells of patients suffering from mucopolysaccharidoses, acts by influencing an epidermal growth factor-dependent pathwayJ Biomed Sci2009162619272193