Literature DB >> 21090963

The Fabry cardiomyopathy: models for the cardiologist.

Frank Weidemann1, Markus Niemann, David G Warnock, Georg Ertl, Christoph Wanner.   

Abstract

Fabry disease is an X-linked lysosomal storage disorder caused by α-galactosidase A deficiency. Intracellular accumulation of globotriaosylceramide starts in utero and progressively develops in various tissues and organs. Cardiac involvement is frequent, and its presentation as concentric nonobstructive left ventricular hypertrophy serves as a model for other hypertrophic cardiomyopathies. This review describes the Fabry cardiomyopathy, its treatment, and multidisciplinary patient care models. These models will help clinicians in diagnosing, assessing, and treating patients with Fabry disease. As the models can be extrapolated to other diseases, they might contribute to more optimal clinical management of patients with other cardiac disorders.

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Year:  2011        PMID: 21090963     DOI: 10.1146/annurev-med-090910-085119

Source DB:  PubMed          Journal:  Annu Rev Med        ISSN: 0066-4219            Impact factor:   13.739


  12 in total

Review 1.  Clinical Spectrum of PRKAG2 Syndrome.

Authors:  Andrea Giuseppe Porto; Francesca Brun; Giovanni Maria Severini; Pasquale Losurdo; Enrico Fabris; Matthew R G Taylor; Luisa Mestroni; Gianfranco Sinagra
Journal:  Circ Arrhythm Electrophysiol       Date:  2016-01

2.  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

Review 3.  The Genetic Challenges and Opportunities in Advanced Heart Failure.

Authors:  Fady Hannah-Shmouni; Sara B Seidelmann; Sandra Sirrs; Arya Mani; Daniel Jacoby
Journal:  Can J Cardiol       Date:  2015-08-21       Impact factor: 5.223

4.  Clinically relevant functional annotation of genotype.

Authors:  Calum A MacRae; Ramachandran S Vasan
Journal:  Circ Cardiovasc Genet       Date:  2014-02

5.  Arrhythmias in Fabry cardiomyopathy.

Authors:  Deepak Acharya; Peter Robertson; G Neal Kay; Leslie Jackson; David G Warnock; Vance J Plumb; José A Tallaj
Journal:  Clin Cardiol       Date:  2012-08-09       Impact factor: 2.882

Review 6.  MicroRNAs in hypertrophic cardiomyopathy: pathogenesis, diagnosis, treatment potential and roles as clinical biomarkers.

Authors:  Fanyan Luo; Wei Liu; Haisong Bu
Journal:  Heart Fail Rev       Date:  2022-03-25       Impact factor: 4.654

Review 7.  Diagnosis and management of hypertrophic cardiomyopathy.

Authors:  Antonis Pantazis; Annina S Vischer; Maria Carrillo Perez-Tome; Silvia Castelletti
Journal:  Echo Res Pract       Date:  2015-03-11

8.  Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.

Authors:  Derralynn A Hughes; Kathleen Nicholls; Suma P Shankar; Gere Sunder-Plassmann; David Koeller; Khan Nedd; Gerard Vockley; Takashi Hamazaki; Robin Lachmann; Toya Ohashi; Iacopo Olivotto; Norio Sakai; Patrick Deegan; David Dimmock; François Eyskens; Dominique P Germain; Ozlem Goker-Alpan; Eric Hachulla; Ana Jovanovic; Charles M Lourenco; Ichiei Narita; Mark Thomas; William R Wilcox; Daniel G Bichet; Raphael Schiffmann; Elizabeth Ludington; Christopher Viereck; John Kirk; Julie Yu; Franklin Johnson; Pol Boudes; Elfrida R Benjamin; David J Lockhart; Carrolee Barlow; Nina Skuban; Jeffrey P Castelli; Jay Barth; Ulla Feldt-Rasmussen
Journal:  J Med Genet       Date:  2016-11-10       Impact factor: 6.318

9.  Cardiomyopathy and response to enzyme replacement therapy in a male mouse model for Fabry disease.

Authors:  Aurelie Nguyen Dinh Cat; Brigitte Escoubet; Vincent Agrapart; Violaine Griol-Charhbili; Trenton Schoeb; Wenguang Feng; Edgar Jaimes; David G Warnock; Frederic Jaisser
Journal:  PLoS One       Date:  2012-05-04       Impact factor: 3.240

Review 10.  Fibrosis: a key feature of Fabry disease with potential therapeutic implications.

Authors:  Frank Weidemann; Maria D Sanchez-Niño; Juan Politei; João-Paulo Oliveira; Christoph Wanner; David G Warnock; Alberto Ortiz
Journal:  Orphanet J Rare Dis       Date:  2013-08-06       Impact factor: 4.123

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