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For: Giugliani R, Lampe C, Guffon N, Ketteridge D, Leão-Teles E, Wraith JE, Jones SA, Piscia-Nichols C, Lin P, Quartel A, Harmatz P. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A 2014;164A:1953-64. [PMID: 24764221 DOI: 10.1002/ajmg.a.36584] [Cited by in Crossref: 57] [Cited by in F6Publishing: 46] [Article Influence: 7.1] [Reference Citation Analysis]
Number Citing Articles
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12 Tomanin R, Karageorgos L, Zanetti A, Al-Sayed M, Bailey M, Miller N, Sakuraba H, Hopwood JJ. Mucopolysaccharidosis type VI (MPS VI) and molecular analysis: Review and classification of published variants in the ARSB gene. Hum Mutat 2018;39:1788-802. [PMID: 30118150 DOI: 10.1002/humu.23613] [Cited by in Crossref: 16] [Cited by in F6Publishing: 15] [Article Influence: 4.0] [Reference Citation Analysis]
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14 Burton BK, Jego V, Mikl J, Jones SA. Survival in idursulfase-treated and untreated patients with mucopolysaccharidosis type II: data from the Hunter Outcome Survey (HOS). J Inherit Metab Dis 2017;40:867-74. [PMID: 28887757 DOI: 10.1007/s10545-017-0075-x] [Cited by in Crossref: 27] [Cited by in F6Publishing: 20] [Article Influence: 5.4] [Reference Citation Analysis]
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17 İnci A, Okur İ, Tümer L, Biberoğlu G, Öktem M, Ezgü F. Clinical and event-based outcomes of patients with mucopolysaccharidosis VI receiving enzyme replacement therapy in Turkey: a case series. Orphanet J Rare Dis 2021;16:438. [PMID: 34666789 DOI: 10.1186/s13023-021-02060-4] [Reference Citation Analysis]
18 Ferla R, Claudiani P, Savarese M, Kozarsky K, Parini R, Scarpa M, Donati MA, Sorge G, Hopwood JJ, Parenti G, Fecarotta S, Nigro V, Sivri HS, Van Der Ploeg A, Andria G, Brunetti-Pierri N, Auricchio A. 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. Hum Gene Ther 2015;26:145-52. [PMID: 25654180 DOI: 10.1089/hum.2014.109] [Cited by in Crossref: 12] [Cited by in F6Publishing: 10] [Article Influence: 1.7] [Reference Citation Analysis]
19 Treadwell M, Harmatz PR, Burton BK, Mitchell JJ, Muschol N, Jones SA, Pastores GM, Lau HA, Sparkes R, Sutton VR, Meesen B, Haller CA, Shaywitz AJ, Gold JI. Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study. Journal of Inborn Errors of Metabolism and Screening 2017;5:232640981771885. [DOI: 10.1177/2326409817718850] [Cited by in Crossref: 4] [Cited by in F6Publishing: 1] [Article Influence: 0.8] [Reference Citation Analysis]
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21 Guio JA, Adolfogiraldo-ospina G. Impact of Enzyme Replacement Therapy in a Patient Younger Than 2 Years Diagnosed With Maroteaux-Lamy Syndrome (MPS VI). Journal of Inborn Errors of Metabolism and Screening 2017;5:232640981771884. [DOI: 10.1177/2326409817718849] [Reference Citation Analysis]
22 Jurecka A, Tylki-szymańska A. Enzyme replacement therapy: lessons learned and emerging questions. Expert Opinion on Orphan Drugs 2015;3:293-305. [DOI: 10.1517/21678707.2015.1017469] [Cited by in Crossref: 14] [Cited by in F6Publishing: 8] [Article Influence: 2.0] [Reference Citation Analysis]
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24 Leiro B, Phillips D, Duiker M, Harmatz P, Charles S. Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome): defining and measuring functional impacts in pediatric patients. Orphanet J Rare Dis 2021;16:500. [PMID: 34857033 DOI: 10.1186/s13023-021-02113-8] [Reference Citation Analysis]
25 D'Avanzo F, Zanetti A, De Filippis C, Tomanin R. Mucopolysaccharidosis Type VI, an Updated Overview of the Disease. Int J Mol Sci 2021;22:13456. [PMID: 34948256 DOI: 10.3390/ijms222413456] [Reference Citation Analysis]
26 Maegawa GHB. Lysosomal Leukodystrophies Lysosomal Storage Diseases Associated With White Matter Abnormalities. J Child Neurol 2019;34:339-58. [PMID: 30757954 DOI: 10.1177/0883073819828587] [Cited by in Crossref: 6] [Cited by in F6Publishing: 5] [Article Influence: 2.0] [Reference Citation Analysis]
27 Brunelli MJ, Atallah ÁN, da Silva EM. Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI. Cochrane Database Syst Rev 2016;3:CD009806. [PMID: 26943923 DOI: 10.1002/14651858.CD009806.pub2] [Cited by in Crossref: 8] [Cited by in F6Publishing: 10] [Article Influence: 1.3] [Reference Citation Analysis]
28 Kakkis E, Marsden D. Urinary glycosaminoglycans as a potential biomarker for evaluating treatment efficacy in subjects with mucopolysaccharidoses. Molecular Genetics and Metabolism 2020;130:7-15. [DOI: 10.1016/j.ymgme.2020.02.006] [Cited by in Crossref: 2] [Cited by in F6Publishing: 2] [Article Influence: 1.0] [Reference Citation Analysis]
29 Furujo M, Kosuga M, Okuyama T. Enzyme replacement therapy attenuates disease progression in two Japanese siblings with mucopolysaccharidosis type VI: 10-Year follow up. Mol Genet Metab Rep 2017;13:69-75. [PMID: 28983456 DOI: 10.1016/j.ymgmr.2017.08.007] [Cited by in Crossref: 22] [Cited by in F6Publishing: 20] [Article Influence: 4.4] [Reference Citation Analysis]
30 Parini R, Deodato F. Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations. Int J Mol Sci 2020;21:E2975. [PMID: 32340185 DOI: 10.3390/ijms21082975] [Cited by in Crossref: 16] [Cited by in F6Publishing: 15] [Article Influence: 8.0] [Reference Citation Analysis]
31 Solano M, Fainboim A, Politei J, Porras-Hurtado GL, Martins AM, Souza CFM, Koch FM, Amartino H, Satizábal JM, Horovitz DDG, Medeiros PFV, Honjo RS, Lourenço CM. Enzyme replacement therapy interruption in patients with Mucopolysaccharidoses: Recommendations for distinct scenarios in Latin America. Mol Genet Metab Rep 2020;23:100572. [PMID: 32140416 DOI: 10.1016/j.ymgmr.2020.100572] [Cited by in Crossref: 5] [Cited by in F6Publishing: 5] [Article Influence: 2.5] [Reference Citation Analysis]
32 Harmatz P, Hendriksz C, Lampe C, Mcgill J, Parini R, Leão-teles E, Valayannopoulos V, Cole T, Matousek R, Graham S, Guffon N, Quartel A. The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). Molecular Genetics and Metabolism 2017;122:107-12. [DOI: 10.1016/j.ymgme.2017.03.008] [Cited by in Crossref: 20] [Cited by in F6Publishing: 17] [Article Influence: 4.0] [Reference Citation Analysis]
33 Tomatsu S, Pitz S, Hampel U. Ophthalmological Findings in Mucopolysaccharidoses. J Clin Med 2019;8:E1467. [PMID: 31540112 DOI: 10.3390/jcm8091467] [Cited by in Crossref: 13] [Cited by in F6Publishing: 10] [Article Influence: 4.3] [Reference Citation Analysis]
34 Mitchell J, Berger KI, Borgo A, Braunlin EA, Burton BK, Ghotme KA, Kircher SG, Molter D, Orchard PJ, Palmer J, Pastores GM, Rapoport DM, Wang RY, White K. Unique medical issues in adult patients with mucopolysaccharidoses. European Journal of Internal Medicine 2016;34:2-10. [DOI: 10.1016/j.ejim.2016.05.017] [Cited by in Crossref: 18] [Cited by in F6Publishing: 14] [Article Influence: 3.0] [Reference Citation Analysis]
35 Lampe C, Harmatz PR, Parini R, Sharma R, Teles EL, Johnson J, Sivam D, Sisic Z. Enzyme replacement therapy initiated in adulthood: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program. Mol Genet Metab 2019;127:355-60. [PMID: 31324526 DOI: 10.1016/j.ymgme.2019.06.008] [Cited by in Crossref: 4] [Cited by in F6Publishing: 3] [Article Influence: 1.3] [Reference Citation Analysis]
36 Guarany NR, Vanz AP, Wilke MVMB, Bender DD, Borges MD, Giugliani R, Schwartz IVD. Mucopolysaccharidosis: Caregiver Quality of Life. Journal of Inborn Errors of Metabolism and Screening 2015;3:232640981561380. [DOI: 10.1177/2326409815613804] [Cited by in Crossref: 5] [Cited by in F6Publishing: 1] [Article Influence: 0.7] [Reference Citation Analysis]
37 Quartel A, Hendriksz CJ, Parini R, Graham S, Lin P, Harmatz P. Growth Charts for Individuals with Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome). JIMD Rep 2015;18:1-11. [PMID: 25518809 DOI: 10.1007/8904_2014_333] [Cited by in Crossref: 9] [Cited by in F6Publishing: 19] [Article Influence: 1.1] [Reference Citation Analysis]
38 Guffon N, Chowdary P, Teles EL, Hughes D, Hennermann JB, Huot-Marchand P, Faudot-Vernier E, Lacombe O, Fiquet A, Richard MP, Abitbol JL, Tallandier M, Hendriksz CJ. Oral treatment for mucopolysaccharidosis VI: Outcomes of the first phase IIa study with odiparcil. J Inherit Metab Dis 2021. [PMID: 34910312 DOI: 10.1002/jimd.12467] [Reference Citation Analysis]
39 Lin HY, Lee CL, Chang CY, Chiu PC, Chien YH, Niu DM, Tsai FJ, Hwu WL, Lin SJ, Lin JL, Chao MC, Chang TM, Tsai WH, Wang TJ, Chuang CK, Lin SP. Survival and diagnostic age of 175 Taiwanese patients with mucopolysaccharidoses (1985-2019). Orphanet J Rare Dis 2020;15:314. [PMID: 33160388 DOI: 10.1186/s13023-020-01598-z] [Cited by in Crossref: 6] [Cited by in F6Publishing: 5] [Article Influence: 3.0] [Reference Citation Analysis]
40 Schwab ME, Brown JEH, Lianoglou B, Jin C, Conroy PC, Gallagher RC, Harmatz P, Mackenzie TC. Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients. Orphanet J Rare Dis 2022;17. [DOI: 10.1186/s13023-022-02178-z] [Reference Citation Analysis]
41 Stepien KM, Gevorkyan AK, Hendriksz CJ, Lobzhanidze TV, Pérez-López J, Tol G, Del Toro Riera M, Vashakmadze ND, Lampe C. Critical clinical situations in adult patients with Mucopolysaccharidoses (MPS). Orphanet J Rare Dis 2020;15:114. [PMID: 32410642 DOI: 10.1186/s13023-020-01382-z] [Cited by in Crossref: 3] [Cited by in F6Publishing: 3] [Article Influence: 1.5] [Reference Citation Analysis]
42 Solanki GA, Sun PP, Martin KW, Hendriksz CJ, Lampe C, Guffon N, Hung A, Sisic Z, Shediac R, Harmatz PR; CSP Study Group. Cervical cord compression in mucopolysaccharidosis VI (MPS VI): Findings from the MPS VI Clinical Surveillance Program (CSP). Mol Genet Metab 2016;118:310-8. [PMID: 27339555 DOI: 10.1016/j.ymgme.2016.06.001] [Cited by in Crossref: 28] [Cited by in F6Publishing: 19] [Article Influence: 4.7] [Reference Citation Analysis]
43 Wang RY, da Silva Franco JF, López-Valdez J, Martins E, Sutton VR, Whitley CB, Zhang L, Cimms T, Marsden D, Jurecka A, Harmatz P. The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII. Mol Genet Metab 2020;129:219-27. [PMID: 32063397 DOI: 10.1016/j.ymgme.2020.01.003] [Cited by in Crossref: 6] [Cited by in F6Publishing: 6] [Article Influence: 3.0] [Reference Citation Analysis]
44 Harmatz PR, Lampe C, Parini R, Sharma R, Teles EL, Johnson J, Sivam D, Sisic Z. Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program. J Inherit Metab Dis 2019;42:519-26. [PMID: 30834539 DOI: 10.1002/jimd.12079] [Cited by in Crossref: 1] [Cited by in F6Publishing: 3] [Article Influence: 0.3] [Reference Citation Analysis]
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46 Ullal AJ, Millington DS, Bali DS. Development of a fluorometric microtiter plate-based enzyme assay for arylsulfatase B (MPS VI) using dried blood spots. Mol Genet Metab Rep 2014;1:465-7. [PMID: 27896124 DOI: 10.1016/j.ymgmr.2014.10.005] [Cited by in Crossref: 6] [Cited by in F6Publishing: 5] [Article Influence: 0.8] [Reference Citation Analysis]
47 Ferla R, Alliegro M, Marteau JB, Dell'Anno M, Nusco E, Pouillot S, Galimberti S, Valsecchi MG, Zuliani V, Auricchio A. Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI. Mol Ther Methods Clin Dev 2017;6:143-58. [PMID: 28932756 DOI: 10.1016/j.omtm.2017.07.004] [Cited by in Crossref: 17] [Cited by in F6Publishing: 16] [Article Influence: 3.4] [Reference Citation Analysis]
48 Scarpa M, Harmatz PR, Meesen B, Giugliani R. Outcomes of a Physician Survey on the Type, Progression, Assessment, and Treatment of Neurological Disease in Mucopolysaccharidoses. Journal of Inborn Errors of Metabolism and Screening 2018;6:232640981875937. [DOI: 10.1177/2326409818759370] [Cited by in Crossref: 2] [Cited by in F6Publishing: 1] [Article Influence: 0.5] [Reference Citation Analysis]
49 Lin HY, Chuang CK, Wang CH, Chien YH, Wang YM, Tsai FJ, Chou YY, Lin SJ, Pan HP, Niu DM, Hwu WL, Ke YY, Lin SP. Long-term galsulfase enzyme replacement therapy in Taiwanese mucopolysaccharidosis VI patients: A case series. Mol Genet Metab Rep 2016;7:63-9. [PMID: 27134829 DOI: 10.1016/j.ymgmr.2016.04.003] [Cited by in Crossref: 22] [Cited by in F6Publishing: 15] [Article Influence: 3.7] [Reference Citation Analysis]
50 Pires de Mello P, Lopes Barth A, de Araujo Torres D, Pires de Mello Valente M, Dain Gandelman Horovitz D. Laryngeal, Tracheal, and Bronchial Disease in the Mucopolysaccharidoses: Endoscopic Study. Diagnostics (Basel) 2020;10:E37. [PMID: 31936731 DOI: 10.3390/diagnostics10010037] [Cited by in Crossref: 2] [Cited by in F6Publishing: 1] [Article Influence: 1.0] [Reference Citation Analysis]
51 Lampe C, McNelly B, Gevorkian AK, Hendriksz CJ, Lobzhanidze TV, Pérez-López J, Stepien KM, Vashakmadze ND, Del Toro M. Transition of patients with mucopolysaccharidosis from paediatric to adult care. Mol Genet Metab Rep 2019;21:100508. [PMID: 31687335 DOI: 10.1016/j.ymgmr.2019.100508] [Cited by in Crossref: 5] [Cited by in F6Publishing: 4] [Article Influence: 1.7] [Reference Citation Analysis]
52 Shapiro EG, Eisengart JB. The natural history of neurocognition in MPS disorders: A review. Mol Genet Metab 2021;133:8-34. [PMID: 33741271 DOI: 10.1016/j.ymgme.2021.03.002] [Cited by in Crossref: 3] [Cited by in F6Publishing: 4] [Article Influence: 3.0] [Reference Citation Analysis]
53 Pineda M, O'Callaghan M, Fernandez Lopez A, Coll MJ, Ullot R, Garcia-Fructuoso G. Clinical Evolution After Enzyme Replacement Therapy in Twins with the Severe Form of Maroteaux-Lamy Syndrome. JIMD Rep 2016;30:7-14. [PMID: 26920906 DOI: 10.1007/8904_2016_530] [Cited by in Crossref: 1] [Cited by in F6Publishing: 1] [Article Influence: 0.2] [Reference Citation Analysis]
54 Wolfberg J, Chintalapati K, Tomatsu S, Nagao K. Hearing Loss in Mucopolysaccharidoses: Current Knowledge and Future Directions. Diagnostics (Basel) 2020;10:E554. [PMID: 32759694 DOI: 10.3390/diagnostics10080554] [Cited by in Crossref: 2] [Cited by in F6Publishing: 1] [Article Influence: 1.0] [Reference Citation Analysis]
55 Horovitz DDG, Leão EKEA, Ribeiro EM, Martins AM, Barth AL, Neri JICF, Kerstenetzky M, Siqueira ACM, Ribeiro BFR, Kim CA, Santos FC, Franco JFS, Lichtvan LCL, Giuliani LR, Rodrigues MDCS, Bonatti RCF, Teixeira TB, Gonçalves A, Lourenço CM, Pereira ASS, Acosta AX. Long-term impact of early initiation of enzyme replacement therapy in 34 MPS VI patients: A resurvey study. Mol Genet Metab 2021;133:94-9. [PMID: 33678523 DOI: 10.1016/j.ymgme.2021.02.006] [Reference Citation Analysis]