Literature DB >> 16601901

Effect of discontinuing of laronidase in a patient with mucopolysaccharidosis type I.

A T Anbu1, J Mercer, J E Wraith.   

Abstract

We present a patient on enzyme replacement therapy who showed rapid deterioration when laronidase was discontinued owing to pregnancy.

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Year:  2006        PMID： 16601901     DOI： 10.1007/s10545-006-0237-8

Source DB:  PubMed          Journal:  J Inherit Metab Dis        ISSN： 0141-8955            Impact factor:   4.982


  3 in total

1.  Enzyme-replacement therapy in mucopolysaccharidosis I.

Authors:  E D Kakkis; J Muenzer; G E Tiller; L Waber; J Belmont; M Passage; B Izykowski; J Phillips; R Doroshow; I Walot; R Hoft; E F Neufeld
Journal:  N Engl J Med       Date:  2001-01-18       Impact factor: 91.245

2.  Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).

Authors:  James E Wraith; Lorne A Clarke; Michael Beck; Edwin H Kolodny; Gregory M Pastores; Joseph Muenzer; David M Rapoport; Kenneth I Berger; Stuart J Swiedler; Emil D Kakkis; Tanja Braakman; Elenie Chadbourne; Karen Walton-Bowen; Gerald F Cox
Journal:  J Pediatr       Date:  2004-05       Impact factor: 4.406

3.  Pregnancies in Gaucher disease: a 5-year study.

Authors:  Yonatan Elstein; Vered Eisenberg; Sorina Granovsky-Grisaru; Ron Rabinowitz; Arnon Samueloff; Ari Zimran; Deborah Elstein
Journal:  Am J Obstet Gynecol       Date:  2004-02       Impact factor: 8.661
  3 in total
  8 in total

Review 1.  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

2.  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

3.  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 4.  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

5.  Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships.

Authors:  Nouriya A Al-Sannaa; Luisa Bay; Deborah S Barbouth; Youssef Benhayoun; Cyril Goizet; Norberto Guelbert; Simon A Jones; Sandra Obikawa Kyosen; Ana Maria Martins; Chanika Phornphutkul; Celia Reig; Rebecca Pleat; Shari Fallet; Iva Ivanovska Holder
Journal:  Orphanet J Rare Dis       Date:  2015-10-07       Impact factor: 4.123

6.  Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series.

Authors:  Dafne Dain Gandelman Horovitz; Angelina X Acosta; Roberto Giugliani; Anna Hlavatá; Katarína Hlavatá; Michel C Tchan; Anneliese Lopes Barth; Laercio Cardoso; Emília Katiane Embiruçu de Araújo Leão; Ana Carolina Esposito; Sandra Obikawa Kyosen; Carolina Fischinger Moura De Souza; Ana Maria Martins
Journal:  Orphanet J Rare Dis       Date:  2016-04-29       Impact factor: 4.123

7.  Pregnancy in patients with mucopolysaccharidosis: a case series.

Authors:  Fiona J Stewart; Andrew Bentley; Barbara K Burton; Nathalie Guffon; Susan L Hale; Paul R Harmatz; Susanne G Kircher; Pavan K Kochhar; John J Mitchell; Ursula Plöckinger; Sue Graham; Stephen Sande; Zlatko Sisic; Tracey A Johnston
Journal:  Mol Genet Metab Rep       Date:  2016-08-29

8.  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 in total

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