Literature DB >> 19533640

Enzyme replacement therapy in adult-onset glycogenosis II: is quantitative muscle MRI helpful?

A Pichiecchio1, G U Poloni, S Ravaglia, M Ponzio, G Germani, D Maranzana, A Costa, A Repetto, E Tavazzi, C Danesino, A Moglia, S Bastianello.   

Abstract

Although it has been shown that muscle magnetic resonance imaging (MRI) improves the phenotypic characterization of patients with neuromuscular disorders and allows accurate quantification of muscle and adipose tissue distribution, to date quantitative MRI has not been used to assess the therapeutic response in clinical trials of neuromuscular diseases. We discuss quantitative MRI findings after a 6-month course of enzyme replacement therapy administered to nine patients with adult-onset glycogenosis II.

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Year:  2009        PMID： 19533640     DOI： 10.1002/mus.21304

Source DB:  PubMed          Journal:  Muscle Nerve        ISSN： 0148-639X            Impact factor:   3.217


  10 in total

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Journal:  MAGMA       Date:  2017-03-22       Impact factor: 2.310

2.  Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease.

Authors:  Thomas Hundsberger; Marianne Rohrbach; Lukas Kern; Kai M Rösler
Journal:  J Neurol       Date:  2013-06-08       Impact factor: 4.849

3.  Changes in skeletal muscle qualities during enzyme replacement therapy in late-onset type II glycogenosis: temporal and spatial pattern of mass vs. strength response.

Authors:  Sabrina Ravaglia; Anna Pichiecchio; Michela Ponzio; Cesare Danesino; Kolsoum Saeidi Garaghani; Guy Umberto Poloni; Antonio Toscano; Arrigo Moglia; Annalisa Carlucci; Paola Bini; Mauro Ceroni; Stefano Bastianello
Journal:  J Inherit Metab Dis       Date:  2010-09-16       Impact factor: 4.982

4.  Whole-body muscle MRI to detect myopathies in non-extrapyramidal bent spine syndrome.

Authors:  Mickaël Ohana; Marie-Christine Durand; Catherine Marty; Jean-Philippe Lazareth; Thierry Maisonobe; Dominique Mompoint; Robert-Yves Carlier
Journal:  Skeletal Radiol       Date:  2014-05-31       Impact factor: 2.199

5.  Function, structure and quality of striated muscles in the lower extremities in patients with late onset Pompe Disease-an MRI study.

Authors:  Michael Vaeggemose; Rosa Andersen Mencagli; Julie Schjødtz Hansen; Bianca Dräger; Steffen Ringgaard; John Vissing; Henning Andersen
Journal:  PeerJ       Date:  2021-05-06       Impact factor: 2.984

Review 6.  Neuromuscular imaging in inherited muscle diseases.

Authors:  Mike P Wattjes; Rudolf A Kley; Dirk Fischer
Journal:  Eur Radiol       Date:  2010-04-27       Impact factor: 5.315

7.  Muscle MRI Findings in Childhood/Adult Onset Pompe Disease Correlate with Muscle Function.

Authors:  Sebastián Figueroa-Bonaparte; Sonia Segovia; Jaume Llauger; Izaskun Belmonte; Irene Pedrosa; Aída Alejaldre; Mercè Mayos; Guillermo Suárez-Cuartín; Eduard Gallardo; Isabel Illa; Jordi Díaz-Manera
Journal:  PLoS One       Date:  2016-10-06       Impact factor: 3.240

8.  Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy.

Authors:  Sean N Prater; Trusha T Patel; Anne F Buckley; Hanna Mandel; Eugene Vlodavski; Suhrad G Banugaria; Erin J Feeney; Nina Raben; Priya S Kishnani
Journal:  Orphanet J Rare Dis       Date:  2013-06-20       Impact factor: 4.123

9.  Muscle imaging data in late-onset Pompe disease reveal a correlation between the pre-existing degree of lipomatous muscle alterations and the efficacy of long-term enzyme replacement therapy.

Authors:  Kai Michael Gruhn; Christoph Malte Heyer; Anne-Katrin Güttsches; Robert Rehmann; Volkmar Nicolas; Tobias Schmidt-Wilcke; Martin Tegenthoff; Matthias Vorgerd; Rudolf Andre Kley
Journal:  Mol Genet Metab Rep       Date:  2015-04-21

10.  Slow, progressive myopathy in neonatally treated patients with infantile-onset Pompe disease: a muscle magnetic resonance imaging study.

Authors:  Steven Shinn-Forng Peng; Wuh-Liang Hwu; Ni-Chung Lee; Fuu-Jen Tsai; Wen-Hui Tsai; Yin-Hsiu Chien
Journal:  Orphanet J Rare Dis       Date:  2016-05-17       Impact factor: 4.123
  10 in total

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