Literature DB >> 12733069

Enzyme replacement therapy in an adolescent with Fabry disease.

Sabine Illsinger1, Thomas Luecke2, Hendrik Langen2, Anibh M Das2.   

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Year:  2003        PMID： 12733069     DOI： 10.1007/s00431-003-1222-y

Source DB:  PubMed          Journal:  Eur J Pediatr        ISSN： 0340-6199            Impact factor:   3.183


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  5 in total

1.  Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease.

Authors:  C M Eng; N Guffon; W R Wilcox; D P Germain; P Lee; S Waldek; L Caplan; G E Linthorst; R J Desnick
Journal:  N Engl J Med       Date:  2001-07-05       Impact factor: 91.245

2.  Enzymatic defect in Fabry's disease. Ceramidetrihexosidase deficiency.

Authors:  R O Brady; A E Gal; R M Bradley; E Martensson; A L Warshaw; L Laster
Journal:  N Engl J Med       Date:  1967-05-25       Impact factor: 91.245

3.  Enzyme replacement therapy in Fabry disease: a randomized controlled trial.

Authors:  R Schiffmann; J B Kopp; H A Austin; S Sabnis; D F Moore; T Weibel; J E Balow; R O Brady
Journal:  JAMA       Date:  2001-06-06       Impact factor: 56.272

4.  A phase 1/2 clinical trial of enzyme replacement in fabry disease: pharmacokinetic, substrate clearance, and safety studies.

Authors:  C M Eng; M Banikazemi; R E Gordon; M Goldman; R Phelps; L Kim; A Gass; J Winston; S Dikman; J T Fallon; S Brodie; C B Stacy; D Mehta; R Parsons; K Norton; M O'Callaghan; R J Desnick
Journal:  Am J Hum Genet       Date:  2001-02-01       Impact factor: 11.025

Review 5.  Pain assessment: the advantages of using pain scales in lysosomal storage diseases.

Authors:  C S Cleeland
Journal:  Acta Paediatr Suppl       Date:  2002
  5 in total
  3 in total

1.  Monitoring the clinical and biochemical response to enzyme replacement therapy in three children with Fabry disease.

Authors:  Kevin Mills; Ashok Vellodi; Peter Morris; Donald Cooper; Michael Morris; Elisabeth Young; Bryan Winchester
Journal:  Eur J Pediatr       Date:  2004-10       Impact factor: 3.183

Review 2.  Fabry disease in the era of enzyme replacement therapy: a renal perspective.

Authors:  Monique E Cho; Jeffrey B Kopp
Journal:  Pediatr Nephrol       Date:  2004-04-03       Impact factor: 3.714

Review 3.  Recommendations for the diagnosis and management of Fabry disease in pediatric patients: a document from the Rare Diseases Committee of the Brazilian Society of Nephrology (Comdora-SBN).

Authors:  Maria Helena Vaisbich; Luís Gustavo Modelli de Andrade; Cassiano Augusto Braga Silva; Fellype de Carvalho Barreto
Journal:  J Bras Nefrol       Date:  2022 Apr-Jun
  3 in total

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