Literature DB >> 18008181

No justification for very high-dose enzyme therapy for patients with type III Gaucher disease.

Ari Zimran, Deborah Elstein.   

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Year:  2007        PMID： 18008181     DOI： 10.1007/s10545-007-0783-8

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


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  13 in total

Review 1.  Management of neuronopathic Gaucher disease: a European consensus.

Authors:  A Vellodi; B Bembi; T B de Villemeur; T Collin-Histed; A Erikson; E Mengel; A Rolfs; A Tylki-Szymanska
Journal:  J Inherit Metab Dis       Date:  2001-06       Impact factor: 4.982

2.  Low-dose therapy trumps high-dose therapy again in the treatment of Gaucher disease.

Authors:  Ari Zimran; Deborah Elstein; Ernest Beutler
Journal:  Blood       Date:  2006-08-01       Impact factor: 22.113

3.  Consensus recommendations.

Authors:  Ernest Beutler
Journal:  Br J Haematol       Date:  2007-07-26       Impact factor: 6.998

4.  Computerized cognitive testing in patients with type I Gaucher disease: effects of enzyme replacement and substrate reduction.

Authors:  Deborah Elstein; Judith Guedalia; Glen M Doniger; Ely S Simon; Vered Antebi; Yael Arnon; Ari Zimran
Journal:  Genet Med       Date:  2005-02       Impact factor: 8.822

5.  Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis.

Authors:  T Cox; R Lachmann; C Hollak; J Aerts; S van Weely; M Hrebícek; F Platt; T Butters; R Dwek; C Moyses; I Gow; D Elstein; A Zimran
Journal:  Lancet       Date:  2000-04-29       Impact factor: 79.321

6.  Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease.

Authors:  N W Barton; R O Brady; J M Dambrosia; A M Di Bisceglie; S H Doppelt; S C Hill; H J Mankin; G J Murray; R I Parker; C E Argoff
Journal:  N Engl J Med       Date:  1991-05-23       Impact factor: 91.245

Review 7.  Outcome of type III Gaucher disease on enzyme replacement therapy: review of 55 cases.

Authors:  E H Davies; A Erikson; T Collin-Histed; E Mengel; A Tylki-Szymanska; A Vellodi
Journal:  J Inherit Metab Dis       Date:  2007-11-12       Impact factor: 4.982

8.  Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry.

Authors:  Neal J Weinreb; Joel Charrow; Hans C Andersson; Paige Kaplan; Edwin H Kolodny; Pramod Mistry; Gregory Pastores; Barry E Rosenbloom; C Ronald Scott; Rebecca S Wappner; Ari Zimran
Journal:  Am J Med       Date:  2002-08-01       Impact factor: 4.965

9.  Isolated horizontal supranuclear gaze palsy as a marker of severe systemic involvement in Gaucher's disease.

Authors:  M C Patterson; M Horowitz; R B Abel; J N Currie; K T Yu; C Kaneski; J J Higgins; R R O'Neill; P Fedio; A Pikus
Journal:  Neurology       Date:  1993-10       Impact factor: 9.910

10.  Simultaneous preimplantation genetic diagnosis for Tay-Sachs and Gaucher disease.

Authors:  Gheona Altarescu; Barry Brooks; Ehud Margalioth; Talia Eldar Geva; Ephrat Levy-Lahad; Paul Renbaum
Journal:  Reprod Biomed Online       Date:  2007-07       Impact factor: 3.828
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  4 in total

1.  Potential efficacy of enzyme replacement and substrate reduction therapy in three siblings with Gaucher disease type III.

Authors:  J Cox-Brinkman; M J van Breemen; B T van Maldegem; L Bour; W E Donker; C E M Hollak; F A Wijburg; J M F G Aerts
Journal:  J Inherit Metab Dis       Date:  2008-10-15       Impact factor: 4.982

2.  Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease.

Authors:  Mario A Cabrera-Salazar; Matthew Deriso; Scott D Bercury; Lingyun Li; John T Lydon; William Weber; Nilesh Pande; Mandy A Cromwell; Diane Copeland; John Leonard; Seng H Cheng; Ronald K Scheule
Journal:  PLoS One       Date:  2012-08-17       Impact factor: 3.240

3.  Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease.

Authors:  Karen M Ashe; Dinesh Bangari; Lingyun Li; Mario A Cabrera-Salazar; Scott D Bercury; Jennifer B Nietupski; Christopher G F Cooper; Johannes M F G Aerts; Edward R Lee; Diane P Copeland; Seng H Cheng; Ronald K Scheule; John Marshall
Journal:  PLoS One       Date:  2011-06-29       Impact factor: 3.240

4.  CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.

Authors:  John Marshall; Ying Sun; Dinesh S Bangari; Eva Budman; Hyejung Park; Jennifer B Nietupski; Amy Allaire; Mary A Cromwell; Bing Wang; Gregory A Grabowski; John P Leonard; Seng H Cheng
Journal:  Mol Ther       Date:  2016-03-07       Impact factor: 11.454
  4 in total

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