Literature DB >> 17603800

Rapid deterioration of a patient with mucopolysaccharidosis type I during interruption of enzyme replacement therapy.

Grzegorz Wegrzyn1, Anna Tylki-Szymańska, Anna Liberek, Ewa Piotrowska, Joanna Jakóbkiewicz-Banecka, Jolanta Marucha, Barbara Czartoryska, Alicja Wegrzyn.   

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Year:  2007        PMID： 17603800     DOI： 10.1002/ajmg.a.31831

Source DB:  PubMed          Journal:  Am J Med Genet A        ISSN： 1552-4825            Impact factor:   2.802


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  8 in total

1.  Enzyme replacement in neuronal storage disorders in the pediatric population.

Authors:  Erika F Augustine; Jonathan W Mink
Journal:  Curr Treat Options Neurol       Date:  2013-10       Impact factor: 3.598

2.  Genistin-rich soy isoflavone extract in substrate reduction therapy for Sanfilippo syndrome: An open-label, pilot study in 10 pediatric patients.

Authors:  Ewa Piotrowska; Joanna Jakóbkiewicz-Banecka; Anna Tylki-Szymanska; Anna Liberek; Agnieszka Maryniak; Marcelina Malinowska; Barbara Czartoryska; Ewa Puk; Anna Kloska; Tomasz Liberek; Sylwia Baranska; Alicja Wegrzyn; Grzegorz Wegrzyn
Journal:  Curr Ther Res Clin Exp       Date:  2008-04

Review 3.  Treatment of mucopolysaccharidosis type II (Hunter syndrome): results from a systematic evidence review.

Authors:  Linda A Bradley; Hamish R M Haddow; Glenn E Palomaki
Journal:  Genet Med       Date:  2017-05-18       Impact factor: 8.822

4.  Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment.

Authors:  Roberto Giugliani; Andressa Federhen; Maria Verônica Muñoz Rojas; Taiane Vieira; Osvaldo Artigalás; Louise Lapagesse Pinto; Ana Cecília Azevedo; Angelina Acosta; Carmen Bonfim; Charles Marques Lourenço; Chong Ae Kim; Dafne Horovitz; Denize Bonfim; Denise Norato; Diane Marinho; Durval Palhares; Emerson Santana Santos; Erlane Ribeiro; Eugênia Valadares; Fábio Guarany; Gisele Rosone de Lucca; Helena Pimentel; Isabel Neves de Souza; Jordão Correa; José Carlos Fraga; José Eduardo Goes; José Maria Cabral; José Simionato; Juan Llerena; Laura Jardim; Liane Giuliani; Luiz Carlos Santana da Silva; Mara L Santos; Maria Angela Moreira; Marcelo Kerstenetzky; Márcia Ribeiro; Nicole Ruas; Patricia Barrios; Paulo Aranda; Rachel Honjo; Raquel Boy; Ronaldo Costa; Carolina Souza; Flavio F Alcantara; Silvio Gilberto A Avilla; Simone Fagondes; Ana Maria Martins
Journal:  Genet Mol Biol       Date:  2010-12-01       Impact factor: 1.771

5.  CASE SERIES OF PATIENTS UNDER BIWEEKLY TREATMENT WITH LARONIDASE: A REPORT OF A SINGLE CENTER EXPERIENCE.

Authors:  Sandra Obikawa Kyosen; Leny Toma; Helena Bonciani Nader; Marion Coting Braga; Vanessa Gonçalves Pereira; Sueli Canossa; João Bosco Pesquero; Vânia D'Almeida; Ana Maria Martins
Journal:  Rev Paul Pediatr       Date:  2019-05-09

Review 6.  Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations.

Authors:  Rossella Parini; Federica Deodato
Journal:  Int J Mol Sci       Date:  2020-04-23       Impact factor: 5.923

7.  Enzyme replacement therapy interruption in patients with Mucopolysaccharidoses: Recommendations for distinct scenarios in Latin America.

Authors:  MarthaL Solano; Alejandro Fainboim; Juan Politei; Gloria L Porras-Hurtado; Ana Maria Martins; Carolina F Moura Souza; Felipe Mendez Koch; Hernan Amartino; Jose Maria Satizábal; Dafne D G Horovitz; Paula F V Medeiros; Rachel S Honjo; Charles M Lourenço
Journal:  Mol Genet Metab Rep       Date:  2020-02-27

8.  Transcriptomic analyses suggest that mucopolysaccharidosis patients may be less susceptible to COVID-19.

Authors:  Karolina Pierzynowska; Lidia Gaffke; Grzegorz Węgrzyn
Journal:  FEBS Lett       Date:  2020-09-06       Impact factor: 3.864
  8 in total

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