Zobrazeno 1 - 10
of 133
pro vyhledávání: '"GALSULFASE"'
Publikováno v:
Frontiers in Pharmacology, Vol 15 (2024)
BackgroundAssociated with enzyme deficiencies causing glycosaminoglycans (GAGs) accumulation, mucopolysaccharidosis type VI (MPS VI) is lysosomal storage disorder. In the treatment of MPS VI, galsulfase (Naglazyme) is commonly used as an enzyme repla
Externí odkaz:
https://doaj.org/article/b675943deb8046f98d215462905d299d
Autor:
S. S. Timonova, K. A. Smolova, D. T. Zaripova, M. S. Pantyushenko, M. A. Koroleva, R. L. Anisimov, R. A. Khamitov, A. A. Piskunov, V. N. Bade
Publikováno v:
Биопрепараты: Профилактика, диагностика, лечение, Vol 22, Iss 1, Pp 80-93 (2022)
Mucopolysaccharidosis type VI (Maroteaux–Lamy syndrome) is an orphan genetic disease caused by deficiency of the lysosomal enzyme arylsulfatase B (ASB). The need to develop a highly productive cell line for the production of recombinant ASB, is beh
Externí odkaz:
https://doaj.org/article/58915adae86c4684bea3a680aa9abe9c
Autor:
Dmitry V. Ivanov, Anna I. Ostrun, Vladimir M. Kenis, Tatiana V. Markova, Ekaterina Yu. Zakharova
Publikováno v:
Вопросы современной педиатрии, Vol 20, Iss 6s, Pp 602-610 (2021)
Background. Mucopolysaccharidosis type VI (MPS VI, Maroteaux–Lamy syndrome) is rare autosomal-recessive multisystem disease, one of the group of lysosomal storage diseases. The MPS VI pathogenesis is determined by arylsulfatase B enzyme deficiency
Externí odkaz:
https://doaj.org/article/e6e5518aa9704b3695bb4783b93277b9
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-11 (2021)
Abstract Background The objective of this study was to describe clinical manifestations and events of patients with mucopolysaccharidosis (MPS) VI in Turkey who are treated with galsulfase enzyme replacement therapy (ERT). Clinical data of 14 childre
Externí odkaz:
https://doaj.org/article/deefca80a49349b4bca54098dd4f3cb8
Publikováno v:
Clinical Case Reports, Vol 8, Iss 12, Pp 3482-3487 (2020)
Abstract We report a patient with mucopolysaccharidosis type VI, on long‐term enzyme replacement home therapy. Results support the efficacy and safety benefits, with additional advantage of home therapy to minimize the risk of community‐transmitt
Externí odkaz:
https://doaj.org/article/00d692bf80c1412b9570c7fb5c5cfbc2
Autor:
Mehmet Umut Akyol, Tord D. Alden, Hernan Amartino, Jane Ashworth, Kumar Belani, Kenneth I. Berger, Andrea Borgo, Elizabeth Braunlin, Yoshikatsu Eto, Jeffrey I. Gold, Andrea Jester, Simon A. Jones, Cengiz Karsli, William Mackenzie, Diane Ruschel Marinho, Andrew McFadyen, Jim McGill, John J. Mitchell, Joseph Muenzer, Torayuki Okuyama, Paul J. Orchard, Bob Stevens, Sophie Thomas, Robert Walker, Robert Wynn, Roberto Giugliani, Paul Harmatz, Christian Hendriksz, Maurizio Scarpa, MPS Consensus Programme Steering Committee, MPS Consensus Programme Co-Chairs
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 14, Iss 1, Pp 1-21 (2019)
Abstract Introduction Mucopolysaccharidosis (MPS) VI or Maroteaux-Lamy syndrome (253200) is an autosomal recessive lysosomal storage disorder caused by deficiency in N-acetylgalactosamine-4-sulfatase (arylsulfatase B). The heterogeneity and progressi
Externí odkaz:
https://doaj.org/article/4b4cdd98c75840e4a9c116d9983d5417
Autor:
Liliia A. Osipova, Ludmila M. Kuzenkova, Leyla S. Namazova-Baranova, Anait K. Gevorkyan, Tatiana V. Podkletnova, Nikolay A. Mayanskiy, Grigoriy V. Revunenkov, Nato D. Vashakmadze
Publikováno v:
Вопросы современной педиатрии, Vol 17, Iss 1, Pp 76-84 (2018)
Background. There are limited data on the efficacy of long-term enzyme replacement therapy (ERT) in children with mucopolysaccharidosis (MPS).Objective. Our aim was to study the efficacy and safety of long-term ERT in children with MPS type I, II, an
Externí odkaz:
https://doaj.org/article/e48e052d9bcd427fa28f2326de88a26c
Akademický článek
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Publikováno v:
Molecular Genetics and Metabolism Reports, Vol 13, Iss C, Pp 69-75 (2017)
Early initiation of enzyme replacement therapy (ERT) has demonstrated clinical benefit in patients with mucopolysaccharidosis type VI (MPS VI), a progressive, multisystem autosomal recessive lysosomal disorder caused by N-acetylgalactosamine-4-sulpha
Externí odkaz:
https://doaj.org/article/f8e6e45c385946bfaa4e34ef15cca514
Akademický článek
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