Literature DB >> 20040314

Enzyme replacement therapy for the management of the mucopolysaccharidoses.

J E Wraith1.   

Abstract

Enzyme replacement therapy (ERT) is now available for several of the mucopolysaccharidosis disorders. This brief review summarizes the role of ERT in reducing the burden of peripheral disease in many patients with mucopolysaccharidosis disorders, and describes the challenges that remain in treating the neurological manifestations of these conditions.

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Year:  2009        PMID： 20040314     DOI： 10.5414/cpp47063

Source DB:  PubMed          Journal:  Int J Clin Pharmacol Ther        ISSN： 0946-1965            Impact factor:   1.366


  9 in total

1.  Plasmatic and Urinary Glycosaminoglycans Characterization in Mucopolysaccharidosis II Patient Treated with Enzyme-Replacement Therapy with Idursulfase.

Authors:  Giovanni V Coppa; Dania Buzzega; Lucia Zampini; Francesca Maccari; Lucia Santoro; Fabio Galeotti; Tiziana Galeazzi; Orazio Gabrielli; Nicola Volpi
Journal:  JIMD Rep       Date:  2011-11-04

2.  Pain: a prevalent feature in patients with mucopolysaccharidosis. Results of a cross-sectional national survey.

Authors:  Marion M G Brands; Deniz Güngör; Johanna M P van den Hout; Francois P J Karstens; Esmee Oussoren; Iris Plug; Jaap Jan Boelens; Peter M van Hasselt; Carla E M Hollak; Margot F Mulder; Estela Rubio Gozalbo; Jan A Smeitink; G Peter A Smit; Frits A Wijburg; Hanka Meutgeert; Ans T van der Ploeg
Journal:  J Inherit Metab Dis       Date:  2014-07-22       Impact factor: 4.982

Review 3.  Newborn screening and diagnosis of mucopolysaccharidoses.

Authors:  Shunji Tomatsu; Tadashi Fujii; Masaru Fukushi; Toshihiro Oguma; Tsutomu Shimada; Miho Maeda; Kazuhiro Kida; Yuniko Shibata; Hideyuki Futatsumori; Adriana M Montaño; Robert W Mason; Seiji Yamaguchi; Yasuyuki Suzuki; Tadao Orii
Journal:  Mol Genet Metab       Date:  2013-06-21       Impact factor: 4.797

4.  A genetic model of substrate reduction therapy for mucopolysaccharidosis.

Authors:  William C Lamanna; Roger Lawrence; Stéphane Sarrazin; Carlos Lameda-Diaz; Philip L S M Gordts; Kelley W Moremen; Jeffrey D Esko
Journal:  J Biol Chem       Date:  2012-09-05       Impact factor: 5.157

5.  Plasmatic kinetics of dermatan sulfate during enzyme replacement therapy with iduronate-2-sulfatase in a mucopolysaccharidosis II patient.

Authors:  Nicola Volpi; Lucia Zampini; Francesca Maccari; Lucia Santoro; Fabio Galeotti; Tiziana Galeazzi; Orazio Gabrielli; Giovanni V Coppa
Journal:  Glycoconj J       Date:  2013-03-20       Impact factor: 2.916

6.  A selective screening program for the early detection of mucopolysaccharidosis: Results of the FIND project - a 2-year follow-up study.

Authors:  Cristóbal Colón; J Victor Alvarez; Cristina Castaño; Luís G Gutierrez-Solana; Ana M Marquez; María O'Callaghan; Félix Sánchez-Valverde; Carmen Yeste; María-Luz Couce
Journal:  Medicine (Baltimore)       Date:  2017-05       Impact factor: 1.889

7.  Prevention of lysosomal storage diseases and derivation of mutant stem cell lines by preimplantation genetic diagnosis.

Authors:  Gheona Altarescu; Rachel Beeri; Rachel Eiges; Silvina Epsztejn-Litman; Talia Eldar-Geva; Deborah Elstein; Ari Zimran; Ehud J Margalioth; Ephrat Levy-Lahad; Paul Renbaum
Journal:  Mol Biol Int       Date:  2012-12-26

8.  Synthetic genistein derivatives as modulators of glycosaminoglycan storage.

Authors:  Anna Kloska; Magdalena Narajczyk; Joanna Jakóbkiewicz-Banecka; Grzegorz Grynkiewicz; Wiesław Szeja; Magdalena Gabig-Cimińska; Grzegorz Węgrzyn
Journal:  J Transl Med       Date:  2012-07-30       Impact factor: 5.531

9.  NDST1 Preferred Promoter Confirmation and Identification of Corresponding Transcriptional Inhibitors as Substrate Reduction Agents for Multiple Mucopolysaccharidosis Disorders.

Authors:  Ilona Tkachyova; Xiaolian Fan; Anne-Marie LamHonWah; Bohdana Fedyshyn; Ingrid Tein; Don J Mahuran; Andreas Schulze
Journal:  PLoS One       Date:  2016-09-22       Impact factor: 3.240
  9 in total

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