Title: Low‐frequency enzyme replacement therapy in late‐onset pompe disease
Abstract: Muscle & NerveVolume 47, Issue 4 p. 612-613 Letter to the Editor Low-frequency enzyme replacement therapy in late-onset pompe disease Dar-Shong Lin MD, Corresponding Author Dar-Shong Lin MD Department of Pediatrics, Mackay Memorial Hospital, Taipei, Taiwan Department of Medical Research, Mackay Memorial Hospital, Taipei, Taiwan Mackay Medicine, Nursing and Management College, Taipei, Taiwan Department of Chemical Engineering and Biotechnology, National Taipei University of Technology, Taipei, Taiwan Dar-Shong Lin, Department of Pediatrics, Mackay Memorial Hospital, TaipeiTaiwan Hsuan-Liang Liu, Department of Medical Research, Mackay Memorial Hospital, TaipeiTaiwanSearch for more papers by this authorMing-Fu Chiang PhD, Ming-Fu Chiang PhD Department of Neurosurgery, Mackay Memorial Hospital, Taipei, Taiwan Institute of Injury Prevention and Control, Taipei Medical University, Taipei, TaiwanSearch for more papers by this authorChe-Sheng Ho MD, Che-Sheng Ho MD Department of Pediatrics, Mackay Memorial Hospital, Taipei, TaiwanSearch for more papers by this authorChung-Der Hsiao PhD, Chung-Der Hsiao PhD Department of Bioscience Technology, Chung Yuan Christian University, Chung Li, TaiwanSearch for more papers by this authorChang-Yi Lin MD, Chang-Yi Lin MD Department of Internal Medicine, Mackay Memorial Hospital, Taipei, TaiwanSearch for more papers by this authorNien-Lu Wang PhD, Nien-Lu Wang PhD Department of Pediatric Surgery, Mackay Memorial Hospital, Taipei, TaiwanSearch for more papers by this authorChih-Kuang Chuang BSc, Chih-Kuang Chuang BSc Department of Medical Research, Mackay Memorial Hospital, Taipei, Taiwan Department of Chemical Engineering and Biotechnology, National Taipei University of Technology, Taipei, TaiwanSearch for more papers by this authorYu-Wen Huang BSc, Yu-Wen Huang BSc Department of Medical Research, Mackay Memorial Hospital, Taipei, TaiwanSearch for more papers by this authorPo-Chun Chang BSc, Po-Chun Chang BSc Department of Information Management, Mackay Memorial Hospital, Taipei, TaiwanSearch for more papers by this authorHsuan-Liang Liu PhD, Corresponding Author Hsuan-Liang Liu PhD Department of Chemical Engineering and Biotechnology, National Taipei University of Technology, Taipei, Taiwan Dar-Shong Lin, Department of Pediatrics, Mackay Memorial Hospital, TaipeiTaiwan Hsuan-Liang Liu, Department of Medical Research, Mackay Memorial Hospital, TaipeiTaiwanSearch for more papers by this author Dar-Shong Lin MD, Corresponding Author Dar-Shong Lin MD Department of Pediatrics, Mackay Memorial Hospital, Taipei, Taiwan Department of Medical Research, Mackay Memorial Hospital, Taipei, Taiwan Mackay Medicine, Nursing and Management College, Taipei, Taiwan Department of Chemical Engineering and Biotechnology, National Taipei University of Technology, Taipei, Taiwan Dar-Shong Lin, Department of Pediatrics, Mackay Memorial Hospital, TaipeiTaiwan Hsuan-Liang Liu, Department of Medical Research, Mackay Memorial Hospital, TaipeiTaiwanSearch for more papers by this authorMing-Fu Chiang PhD, Ming-Fu Chiang PhD Department of Neurosurgery, Mackay Memorial Hospital, Taipei, Taiwan Institute of Injury Prevention and Control, Taipei Medical University, Taipei, TaiwanSearch for more papers by this authorChe-Sheng Ho MD, Che-Sheng Ho MD Department of Pediatrics, Mackay Memorial Hospital, Taipei, TaiwanSearch for more papers by this authorChung-Der Hsiao PhD, Chung-Der Hsiao PhD Department of Bioscience Technology, Chung Yuan Christian University, Chung Li, TaiwanSearch for more papers by this authorChang-Yi Lin MD, Chang-Yi Lin MD Department of Internal Medicine, Mackay Memorial Hospital, Taipei, TaiwanSearch for more papers by this authorNien-Lu Wang PhD, Nien-Lu Wang PhD Department of Pediatric Surgery, Mackay Memorial Hospital, Taipei, TaiwanSearch for more papers by this authorChih-Kuang Chuang BSc, Chih-Kuang Chuang BSc Department of Medical Research, Mackay Memorial Hospital, Taipei, Taiwan Department of Chemical Engineering and Biotechnology, National Taipei University of Technology, Taipei, TaiwanSearch for more papers by this authorYu-Wen Huang BSc, Yu-Wen Huang BSc Department of Medical Research, Mackay Memorial Hospital, Taipei, TaiwanSearch for more papers by this authorPo-Chun Chang BSc, Po-Chun Chang BSc Department of Information Management, Mackay Memorial Hospital, Taipei, TaiwanSearch for more papers by this authorHsuan-Liang Liu PhD, Corresponding Author Hsuan-Liang Liu PhD Department of Chemical Engineering and Biotechnology, National Taipei University of Technology, Taipei, Taiwan Dar-Shong Lin, Department of Pediatrics, Mackay Memorial Hospital, TaipeiTaiwan Hsuan-Liang Liu, Department of Medical Research, Mackay Memorial Hospital, TaipeiTaiwanSearch for more papers by this author First published: 30 August 2012 https://doi.org/10.1002/mus.23658Citations: 2Read the full textAboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onEmailFacebookTwitterLinkedInRedditWechat No abstract is available for this article. References 1Furusawa Y, Mori-Yoshimura M, Yamamoto T, Sakamoto C, Wakita M, Kobayashi Y, et al. Effects of enzyme replacement therapy on five patients with advanced late-onset glycogen storage disease type II: a 2-year follow-up study. J Inherit Metab Dis 2012; 35: 301–310. 2Regnery C, Kornblum C, Hanisch F, Vielhaber S, Strigl-Pill N, Grunert B, et al. 36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy. J Inherit Metab Dis 2012; 35: 837–845. 3Vielhaber S, Brejova A, Debska-Vielhaber G, Kaufmann J, Feistner H, Schoenfeld MA, et al. 24-months results in two adults with Pompe disease on enzyme replacement therapy. Clin Neurol Neurosurg 2011; 113: 350–357. 4Bembi B, Pisa FE, Confalonieri M, Ciana G, Fiumara A, Parini R, et al. Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II. J Inherit Metab Dis 2010; 33: 727–735. 5Ravaglia S, Pichiecchio A, Ponzio M, Danesino C, Saeidi Garaghani K, Poloni GU, et al. Changes in skeletal muscle qualities during enzyme replacement therapy in late-onset type II glycogenosis: temporal and spatial pattern of mass vs. strength response. J Inherit Metab Dis 2010; 33: 737–745. 6van Capelle CI, Winkel LP, Hagemans ML, Shapira SK, Arts WF, van Doorn PA, et al. Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease. Neuromuscul Disord 2008; 18: 447–452. 7Case LE, Koeberl DD, Young SP, Bali D, DeArmey SM, Mackey J, et al. Improvement with ongoing enzyme replacement therapy in advanced late-onset Pompe disease: a case study. Mol Genet Metab 2008; 95: 233–235. 8van der Ploeg AT, Clemens PR, Corzo D, Escolar DM, Florence J, Groeneveld GJ, et al. A randomized study of alglucosidase alfa in late-onset Pompe's disease. N Engl J Med 2010; 362: 1396–1406. 9van der Beek NA, Hagemans ML, Reuser AJ, Hop WC, van der Ploeg AT, van Doorn PA, et al. Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease. Neuromuscul Disord 2009; 19: 113–117. 10Wokke JH, Escolar DM, Pestronk A, Jaffe KM, Carter GT, van den Berg LH, et al. Clinical features of late-onset Pompe disease: a prospective cohort study. Muscle Nerve 2008; 38: 1236–1245. 11Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, et al. Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease. Neurology 2007; 68: 99–109. 12Winkel LP, van den Hout JM, Kamphoven JH, Disseldorp JA, Remmerswaal M, Arts WF, et al. Enzyme replacement therapy in late-onset Pompe's disease: a three-year follow-up. Ann Neurol 2004; 55: 495–502. 13Cupler EJ, Berger KI, Leshner RT, Wolfe GI, Han JJ, Barohn RJ, et al. Consensus treatment recommendations for late-onset Pompe disease. Muscle Nerve 2012; 45: 319–333. Citing Literature Volume47, Issue4April 2013Pages 612-613 ReferencesRelatedInformation
Publication Year: 2013
Publication Date: 2013-01-16
Language: en
Type: letter
Indexed In: ['crossref', 'pubmed']
Access and Citation
Cited By Count: 3
AI Researcher Chatbot
Get quick answers to your questions about the article from our AI researcher chatbot