Literature DB >> 16435211

Mucopolysaccharidosis I under enzyme replacement therapy with laronidase--a mortality case with autopsy report.

H-Y Lin1, S-P Lin, C-K Chuang, M-R Chen, B-F Chen, J E Wraith.   

Abstract

There is little information about MPS I-related complications during laronidase therapy. We describe the first autopsy report of a young male MPS I patient who died of infection-induced cardiopulmonary failure following 2 years of weekly treatment with laronidase.

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Year:  2005        PMID： 16435211     DOI： 10.1007/s10545-005-0211-x

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


  9 in total

1.  Heart weight and heart weight/body weight coefficient in malnourished adults.

Authors:  Daniel Ferreira da Cunha; Selma Freire de Carvalho da Cunha; Marlene Antônia dos Reis; Vicente de Paula Antunes Teixeira
Journal:  Arq Bras Cardiol       Date:  2002-04       Impact factor: 2.000

Review 2.  The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I.

Authors:  James Edward Wraith
Journal:  Expert Opin Pharmacother       Date:  2005-03       Impact factor: 3.889

3.  Cardiovascular changes in mucopolysaccharidoses in Taiwan.

Authors:  Ming-Ren Chen; Shuan-Pei Lin; Haw-Kwei Hwang; Chang-Hsien Yu
Journal:  Acta Cardiol       Date:  2005-02       Impact factor: 1.718

4.  Diagnostic screening for mucopolysaccharidoses by the dimethylmethylene blue method and two dimensional electrophoresis.

Authors:  C K Chuang; S P Lin; S F Chung
Journal:  Zhonghua Yi Xue Za Zhi (Taipei)       Date:  2001-01

5.  Clinical experience in anesthetic management for children with mucopolysaccharidoses: Report of ten cases.

Authors:  Y L Chan; S P Lin; T T Man; C R Cheng
Journal:  Acta Paediatr Taiwan       Date:  2001 Sep-Oct

6.  Mucopolysaccharidosis type I: characterization of novel mutations affecting alpha-L-iduronidase activity.

Authors:  G J Lee-Chen; S P Lin; Y F Tang; Y W Chin
Journal:  Clin Genet       Date:  1999-07       Impact factor: 4.438

7.  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

8.  Mucopolysaccharidosis type I: Identification and characterization of mutations affecting alpha-L-iduronidase activity.

Authors:  Guey-Jen Lee-Chen; Shuan-Pei Lin; I-Shen Chen; Jui-Hung Chang; Chyau-Wen Yang; Yi-Wen Chin
Journal:  J Formos Med Assoc       Date:  2002-06       Impact factor: 3.282

9.  Airway changes in children with mucopolysaccharidoses.

Authors:  Shin-Lin Shih; Y-J Lee; S-P Lin; C-Y Sheu; J G Blickman
Journal:  Acta Radiol       Date:  2002-01       Impact factor: 1.701
  9 in total
  23 in total

1.  Carotid intima-media thickness is increased in patients with mucopolysaccharidoses.

Authors:  Raymond Y Wang; Kelly K Covault; Eileen M Halcrow; Audrey J Gardner; Xiaoling Cao; Robert L Newcomb; Richard D Dauben; Anthony C Chang
Journal:  Mol Genet Metab       Date:  2011-09-10       Impact factor: 4.797

2.  Considering Fabry, but Diagnosing MPS I: Difficulties in the Diagnostic Process.

Authors:  E J Langereis; I E T van den Berg; D J J Halley; B J H M Poorthuis; F M Vaz; J H J Wokke; G E Linthorst
Journal:  JIMD Rep       Date:  2012-10-30

3.  Network Analysis Reveals Proteins Associated with Aortic Dilatation in Mucopolysaccharidoses.

Authors:  Thiago Corrêa; Bruno César Feltes; Esteban Alberto Gonzalez; Guilherme Baldo; Ursula Matte
Journal:  Interdiscip Sci       Date:  2021-01-21       Impact factor: 2.233

4.  Endothelial function in children and adolescents with mucopolysaccharidosis.

Authors:  Aaron S Kelly; Andrea M Metzig; Julia Steinberger; Elizabeth A Braunlin
Journal:  J Inherit Metab Dis       Date:  2012-01-10       Impact factor: 4.982

5.  Carotid intima-media thickness is increased in patients with treated mucopolysaccharidosis types I and II, and correlates with arterial stiffness.

Authors:  Raymond Y Wang; Elizabeth A Braunlin; Kyle D Rudser; Donald R Dengel; Andrea M Metzig; Kelly K Covault; Lynda E Polgreen; Elsa Shapiro; Julia Steinberger; Aaron S Kelly
Journal:  Mol Genet Metab       Date:  2013-11-12       Impact factor: 4.797

6.  Postmortem studies on a patient with mucopolysaccharidosis type I: histopathological findings after one year of enzyme replacement therapy.

Authors:  S Yano; K Moseley; Z Pavlova
Journal:  J Inherit Metab Dis       Date:  2009-03-27       Impact factor: 4.982

Review 7.  Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management.

Authors:  Elizabeth A Braunlin; Paul R Harmatz; Maurizio Scarpa; Beatriz Furlanetto; Christoph Kampmann; James P Loehr; Katherine P Ponder; William C Roberts; Howard M Rosenfeld; Roberto Giugliani
Journal:  J Inherit Metab Dis       Date:  2011-07-09       Impact factor: 4.982

8.  The Murine Model of Mucopolysaccharidosis IIIB Develops Cardiopathies over Time Leading to Heart Failure.

Authors:  Gabriele Giacomo Schiattarella; Giuliana Cerulo; Valeria De Pasquale; Pasquale Cocchiaro; Orlando Paciello; Luigi Avallone; Maria Paola Belfiore; Francesca Iacobellis; Daniele Di Napoli; Fabio Magliulo; Cinzia Perrino; Bruno Trimarco; Giovanni Esposito; Paola Di Natale; Luigi Michele Pavone
Journal:  PLoS One       Date:  2015-07-06       Impact factor: 3.240

9.  A pilot newborn screening program for Mucopolysaccharidosis type I in Taiwan.

Authors:  Shuan-Pei Lin; Hsiang-Yu Lin; Tuen-Jen Wang; Chia-Ying Chang; Chia-Hui Lin; Sung-Fa Huang; Chia-Chen Tsai; Hsuan-Liang Liu; Joan Keutzer; Chih-Kuang Chuang
Journal:  Orphanet J Rare Dis       Date:  2013-09-22       Impact factor: 4.123

10.  Assessment of bone mineral density by dual energy x-ray absorptiometry in patients with mucopolysaccharidoses.

Authors:  Hsiang-Yu Lin; Shou-Chuan Shih; Chih-Kuang Chuang; Ming-Ren Chen; Dau-Ming Niu; Shuan-Pei Lin
Journal:  Orphanet J Rare Dis       Date:  2013-05-11       Impact factor: 4.123
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