Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD)-Reference-Cited by-同舟云学术

Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD)

Published:2017-07-04 Issue:5 Volume:44 Page:449-462
ISSN:0305-1846
Container-title:Neuropathology and Applied Neurobiology
language:en
Short-container-title:Neuropathol Appl Neurobiol

Author:

Ripolone M.¹^ORCID,Violano R.¹,Ronchi D.²,Mondello S.³,Nascimbeni A.⁴,Colombo I.¹,Fagiolari G.¹,Bordoni A.²,Fortunato F.²,Lucchini V.¹,Saredi S.⁵,Filosto M.⁶,Musumeci O.⁷,Tonin P.⁸,Mongini T.⁹,Previtali S.¹⁰,Morandi L.⁵,Angelini C.¹¹,Mora M.⁵,Sandri M.¹²¹³,Sciacco M.¹,Toscano A.⁷,Comi G. P.²,Moggio M.¹

Affiliation:

1. Neuromuscular and Rare Diseases Unit; Department of Neuroscience; Fondazione IRCCS Ca’ Granda; Ospedale Maggiore Policlinico; Milan Italy

2. Neurology Unit; Neuroscience Section; Department of Pathophysiology and Transplantation; Dino Ferrari Centre; IRCCS Foundation Ca’ Granda Ospedale Maggiore Policlinico; University of Milan; Milan Italy

3. Department of Biomedical and Dental Sciences and Morphofunctional Imaging; University of Messina; Messina Italy

4. Department of Neurosciences; University of Padova; Padova Italy

5. Neuromuscular Diseases and Neuroimmunology; Fondazione IRCCS Istituto Neurologico “Carlo Besta”; Milan Italy

6. Unit of Neurology; Center for Neuromuscular Diseases and Neuropathies; University Hospital “Spedali Civili”; Brescia Italy

7. Department of Clinical and Experimental Medicine; Centro di Riferimento Regionale per le Malattie Neuromuscolari rare; University of Messina; Messina Italy

8. Section of Clinical Neurology; Department of Neurological, Biomedical and Movement Sciences; University of Verona; Verona Italy

9. Department of Neurosciences “Rita Levi Montalcini”; University of Turin; Turin Italy

10. Division of Neuroscience; Inspe; San Raffaele; Milan Italy

11. Fondazione San Camillo Hospital IRCCS; Venice Italy

12. Department of Biomedical Science; University of Padova; Padova Italy

13. Dulbecco Telethon Institute at Venetian Institute of Molecular Medicine; Padova Italy

Funder

Fondazione Telethon

Publisher

Wiley

Subject

Physiology (medical),Neurology (clinical),Neurology,Histology,Pathology and Forensic Medicine

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1111/nan.12414/fullpdf

Reference38 articles.

1. Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease);Raben;Curr Mol Med,2002

2. Clinical manifestations and natural course of late-onset Pompe's disease in 54 Dutch patients;Hagemans;Brain,2005

3. Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years;Angelini;J Neurol,2012

4. Intracranial arterial abnormalities in patients with late onset Pompe disease (LOPD);Montagnese;J Inherit Metab Dis,2016

5. Clinical and metabolic correction of Pompe disease by enzyme therapy in acid maltase-deficient quail;Kikuchi;J Clin Invest,1998

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