Literature DB >> 28674962

Fabry disease.

Toshinori Yuasa1, Toshihiro Takenaka2, Koji Higuchi3, Nami Uchiyama3, Yoshihisa Horizoe3, Hideto Cyaen3, Naoko Mizukami4, Kunitsugu Takasaki3, Akira Kisanuki5, Masaaki Miyata3, Mitsuru Ohishi3.   

Abstract

Fabry disease resulting from a deficiency of α-galactosidase A leads to the accumulation of globotriaosylceramide in various organs. Because the disease is an X-linked recessive disorder, males tend to develop more symptoms and more severe symptoms than females. There are also some variants of Fabry disease, and cardiac variant (cardiac Fabry disease) has the dysfunctions only in heart. Cardiac manifestations in Fabry disease are initially symmetrical and concentric left ventricular hypertrophy, and later progressive cardiac dysfunction with localized thinning of the basal posterior wall. In recent years, enzyme replacement therapy has been performed as a treatment for Fabry disease, and the initiation of this therapy is expected before the cardiac fibrosis develops. Therefore, early diagnosis of Fabry disease is essential, and echocardiography is an indispensable tool for clinical practice of this disease. Then, it is necessary to remember this disease as a differential diagnosis when encountering unexplained left ventricular hypertrophy.

Entities:  

Keywords:  Enzyme replacement therapy; Fabry disease; Left ventricular hypertrophy; α-Galactosidase A

Mesh:

Substances:

Year:  2017        PMID: 28674962     DOI: 10.1007/s12574-017-0340-x

Source DB:  PubMed          Journal:  J Echocardiogr        ISSN: 1349-0222


  30 in total

1.  Fabry's disease cardiomyopathy: echocardiographic detection of endomyocardial glycosphingolipid compartmentalization.

Authors:  Maurizio Pieroni; Cristina Chimenti; Francesco De Cobelli; Emanuela Morgante; Alessandro Del Maschio; Carlo Gaudio; Matteo Antonio Russo; Andrea Frustaci
Journal:  J Am Coll Cardiol       Date:  2006-03-29       Impact factor: 24.094

2.  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

3.  Cardiovascular manifestations of Fabry disease: relationships between left ventricular hypertrophy, disease severity, and alpha-galactosidase A activity.

Authors:  Justina C Wu; Carolyn Y Ho; Hicham Skali; Rekha Abichandani; William R Wilcox; Maryam Banikazemi; Seymour Packman; Katherine Sims; Scott D Solomon
Journal:  Eur Heart J       Date:  2010-01-07       Impact factor: 29.983

4.  Early detection of Fabry cardiomyopathy by tissue Doppler imaging.

Authors:  Maurizio Pieroni; Cristina Chimenti; Roberta Ricci; Patrizio Sale; Matteo Antonio Russo; Andrea Frustaci
Journal:  Circulation       Date:  2003-03-31       Impact factor: 29.690

5.  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

6.  An atypical variant of Fabry's disease in men with left ventricular hypertrophy.

Authors:  S Nakao; T Takenaka; M Maeda; C Kodama; A Tanaka; M Tahara; A Yoshida; M Kuriyama; H Hayashibe; H Sakuraba
Journal:  N Engl J Med       Date:  1995-08-03       Impact factor: 91.245

Review 7.  Fabry disease: a review.

Authors:  Charles Masson; Idrissa Cissé; Virginie Simon; Paolo Insalaco; Maurice Audran
Journal:  Joint Bone Spine       Date:  2004-09       Impact factor: 4.929

8.  Fabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome Survey.

Authors:  A Mehta; R Ricci; U Widmer; F Dehout; A Garcia de Lorenzo; C Kampmann; A Linhart; G Sunder-Plassmann; M Ries; M Beck
Journal:  Eur J Clin Invest       Date:  2004-03       Impact factor: 4.686

9.  Long-term effects of enzyme replacement therapy on fabry cardiomyopathy: evidence for a better outcome with early treatment.

Authors:  Frank Weidemann; Markus Niemann; Frank Breunig; Sebastian Herrmann; Meinrad Beer; Stefan Störk; Wolfram Voelker; Georg Ertl; Christoph Wanner; Jörg Strotmann
Journal:  Circulation       Date:  2009-01-19       Impact factor: 29.690

10.  Prevalence of Fabry disease in female patients with late-onset hypertrophic cardiomyopathy.

Authors:  Cristina Chimenti; Maurizio Pieroni; Emanuela Morgante; Daniela Antuzzi; Andrea Russo; Matteo Antonio Russo; Attilio Maseri; Andrea Frustaci
Journal:  Circulation       Date:  2004-08-16       Impact factor: 29.690

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Authors:  I Almeida; S Magalhães; A Nunes
Journal:  Biogerontology       Date:  2021-04-10       Impact factor: 4.277

2.  Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates.

Authors:  Xuling Zhu; Ling Yin; Matt Theisen; Jenny Zhuo; Summar Siddiqui; Becca Levy; Vladimir Presnyak; Andrea Frassetto; Jaclyn Milton; Timothy Salerno; Kerry E Benenato; Joe Milano; Andy Lynn; Staci Sabnis; Kristine Burke; Gilles Besin; Christine M Lukacs; Lin T Guey; Patrick F Finn; Paolo G V Martini
Journal:  Am J Hum Genet       Date:  2019-03-14       Impact factor: 11.025

3.  CARS Imaging Advances Early Diagnosis of Cardiac Manifestation of Fabry Disease.

Authors:  Elen Tolstik; Nairveen Ali; Shuxia Guo; Paul Ebersbach; Dorothe Möllmann; Paula Arias-Loza; Johann Dierks; Irina Schuler; Erik Freier; Jörg Debus; Hideo A Baba; Peter Nordbeck; Thomas Bocklitz; Kristina Lorenz
Journal:  Int J Mol Sci       Date:  2022-05-11       Impact factor: 6.208

4.  Metabolic Disorders and Anesthesia.

Authors:  Cindy Yeoh; Howard Teng; Jacob Jackson; Lee Hingula; Takeshi Irie; Aron Legler; Corrine Levine; Iris Chu; Casey Chai; Luis Tollinche
Journal:  Curr Anesthesiol Rep       Date:  2019-07-12

5.  Quantitative evaluation of retinal and choroidal changes in Fabry disease using optical coherence tomography angiography.

Authors:  Zhongjing Lin; Xiaoxia Pan; Ke Mao; Qin Jiao; Yanwei Chen; Yisheng Zhong; Yu Cheng
Journal:  Lasers Med Sci       Date:  2021-01-06       Impact factor: 3.161

6.  A survey on the patient journey in Fabry disease in Japan.

Authors:  Mina Tsurumi; Asuka Ozaki; Yoshikatsu Eto
Journal:  Mol Genet Metab Rep       Date:  2022-08-17

7.  Plasma lipidomic analysis of sphingolipids in patients with large artery atherosclerosis cerebrovascular disease and cerebral small vessel disease.

Authors:  Qian You; Qing Peng; Zemou Yu; Haiqiang Jin; Jing Zhang; Wei Sun; Yining Huang
Journal:  Biosci Rep       Date:  2020-09-30       Impact factor: 3.840

  7 in total

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