Literature DB >> 21123099

Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT).

Gabor E Linthorst1, Dominique P Germain, Carla E M Hollak, Derralynn Hughes, Arndt Rolfs, Christoph Wanner, Atul Mehta.   

Abstract

The shortage of enzyme for treatment of Fabry disease has caused anxiety among patients, physicians and governments. Following a request from the European Medicines Agency, consensus was reached on the temporary prioritization of patients for treatment based on disease severity and potential reversibility. Advice on follow-up of patients was agreed upon. This consensus is proposed to support the temporary guidelines issued throughout the period of ERT shortage, which will most likely last until April 2011. Copyright Â
© 2010 Elsevier Inc. All rights reserved.

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Year:  2010        PMID： 21123099     DOI： 10.1016/j.ymgme.2010.11.155

Source DB:  PubMed          Journal:  Mol Genet Metab        ISSN： 1096-7192            Impact factor:   4.797


  17 in total

1.  To march in or not to march in.

Authors:  C Allen Black
Journal:  Nat Med       Date:  2011-08-04       Impact factor: 53.440

2.  Effects of switching from agalsidase Beta to agalsidase alfa in 10 patients with anderson-fabry disease.

Authors:  A Pisani; L Spinelli; B Visciano; I Capuano; M Sabbatini; E Riccio; G Messalli; M Imbriaco
Journal:  JIMD Rep       Date:  2012-10-21

3.  Recommendations on reintroduction of agalsidase Beta for patients with fabry disease in europe, following a period of shortage.

Authors:  Gabor E Linthorst; Alessandro P Burlina; Franco Cecchi; Timothy M Cox; Janice M Fletcher; Ulla Feldt-Rasmussen; Roberto Giugliani; Carla E M Hollak; Gunnar Houge; Derralynn Hughes; Iikka Kantola; Robin Lachmann; Monica Lopez; Alberto Ortiz; Rossella Parini; Alberto Rivera; Arndt Rolfs; Uma Ramaswami; Einar Svarstad; Camilla Tondel; Anna Tylki-Szymanska; Bojan Vujkovac; Steven Waldek; Michael West; F Weidemann; Atul Mehta
Journal:  JIMD Rep       Date:  2012-07-14

4.  Agalsidase benefits renal histology in young patients with Fabry disease.

Authors:  Camilla Tøndel; Leif Bostad; Kristin Kampevold Larsen; Asle Hirth; Bjørn Egil Vikse; Gunnar Houge; Einar Svarstad
Journal:  J Am Soc Nephrol       Date:  2013-01       Impact factor: 10.121

5.  Successful management of enzyme replacement therapy in related fabry disease patients with severe adverse events by switching from agalsidase Beta (fabrazyme(®)) to agalsidase alfa (replagal (®)).

Authors:  Kazuya Tsuboi; Hiroshi Yamamoto; Fuji Somura; Hiromi Goto
Journal:  JIMD Rep       Date:  2014-04-10

Review 6.  Contemporary therapeutics and new drug developments for treatment of Fabry disease: a narrative review.

Authors:  Daniel Oder; Jonas Müntze; Peter Nordbeck
Journal:  Cardiovasc Diagn Ther       Date:  2021-04

7.  Patients with Fabry Disease after Enzyme Replacement Therapy Dose Reduction and Switch-2-Year Follow-Up.

Authors:  Malte Lenders; Sima Canaan-Kühl; Johannes Krämer; Thomas Duning; Stefanie Reiermann; Claudia Sommer; Jörg Stypmann; Daniela Blaschke; Nurcan Üçeyler; Hans-Werner Hense; Stefan-Martin Brand; Christoph Wanner; Frank Weidemann; Eva Brand
Journal:  J Am Soc Nephrol       Date:  2015-07-16       Impact factor: 10.121

8.  Patients with Fabry disease after enzyme replacement therapy dose reduction versus treatment switch.

Authors:  Frank Weidemann; Johannes Krämer; Thomas Duning; Malte Lenders; Sima Canaan-Kühl; Alice Krebs; Hans Guerrero González; Claudia Sommer; Nurcan Üçeyler; Markus Niemann; Stefan Störk; Michael Schelleckes; Stefanie Reiermann; Jörg Stypmann; Stefan-Martin Brand; Christoph Wanner; Eva Brand
Journal:  J Am Soc Nephrol       Date:  2014-02-20       Impact factor: 10.121

Review 9.  Fabry Disease: Recognition, Diagnosis, and Treatment of Neurological Features.

Authors:  Michela Ranieri; Gloria Bedini; Eugenio Agostino Parati; Anna Bersano
Journal:  Curr Treat Options Neurol       Date:  2016-07       Impact factor: 3.598

10.  Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain.

Authors:  Saskia M Rombach; Bouwien E Smid; Machtelt G Bouwman; Gabor E Linthorst; Marcel G W Dijkgraaf; Carla E M Hollak
Journal:  Orphanet J Rare Dis       Date:  2013-03-25       Impact factor: 4.123
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