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Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study.
van der Ploeg, Ans; Carlier, Pierre G; Carlier, Robert-Yves; Kissel, John T; Schoser, Benedikt; Wenninger, Stephan; Pestronk, Alan; Barohn, Richard J; Dimachkie, Mazen M; Goker-Alpan, Ozlem; Mozaffar, Tahseen; Pena, Loren D M; Simmons, Zachary; Straub, Volker; Guglieri, Michela; Young, Peter; Boentert, Matthias; Baudin, Pierre-Yves; Wens, Stephan; Shafi, Raheel; Bjartmar, Carl; Thurberg, Beth L.
Affiliation
  • van der Ploeg A; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, Netherlands.
  • Carlier PG; Institut de Myologie, AIM and CEA NMR Laboratory - Spectroscopy Laboratory, Université Pierre et Marie Curie, Paris, France.
  • Carlier RY; Medical Imaging Department, Raymond Poincare University Hospital, Garches, France.
  • Kissel JT; Department of Neurology, Division of Neuromuscular Medicine, Ohio State University Wexner Medical Center, Columbus, OH, USA.
  • Schoser B; Friedrich-Baur-Institut, Neurologische Klinik, Klinikum der Universität München, München, Germany.
  • Wenninger S; Friedrich-Baur-Institut, Neurologische Klinik, Klinikum der Universität München, München, Germany.
  • Pestronk A; Department of Neurology, Washington University School of Medicine, Saint Louis, MO, USA.
  • Barohn RJ; Department of Neurology, University of Kansas Medical Center, Kansas City, KS, USA.
  • Dimachkie MM; Department of Neurology, University of Kansas Medical Center, Kansas City, KS, USA.
  • Goker-Alpan O; Lysosomal Disorders Unit and Center for Clinical Trials, O&O Alpan LLC, Fairfax, VA, USA.
  • Mozaffar T; Department of Neurology, University of California, Irvine, Irvine, CA, USA.
  • Pena LD; Division of Pediatric Medical Genetics, Duke University Medical Center, Durham, NC, USA.
  • Simmons Z; Penn State Hershey Neurology, Hershey, PA, USA.
  • Straub V; Institute of Genetic Medicine, Newcastle University, Newcastle Upon Tyne, United Kingdom.
  • Guglieri M; Institute of Genetic Medicine, Newcastle University, Newcastle Upon Tyne, United Kingdom.
  • Young P; Department of Sleep Medicine and Neuromuscular Disorders, University Hospital of Münster, Münster, Germany.
  • Boentert M; Department of Sleep Medicine and Neuromuscular Disorders, University Hospital of Münster, Münster, Germany.
  • Baudin PY; C.R.I.S., Tournai, France.
  • Wens S; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, Netherlands.
  • Shafi R; Sanofi Genzyme, Cambridge, MA, USA.
  • Bjartmar C; Sanofi Genzyme, Cambridge, MA, USA.
  • Thurberg BL; Sanofi Genzyme, Cambridge, MA, USA. Electronic address: Beth.Thurberg@genzyme.com.
Mol Genet Metab ; 119(1-2): 115-23, 2016 09.
Article in En | MEDLINE | ID: mdl-27473031
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glycogen Storage Disease Type II / Muscle, Skeletal / Alpha-Glucosidases / Enzyme Replacement Therapy Type of study: Clinical_trials / Qualitative_research Limits: Adult / Aged / Female / Humans / Male / Middle aged Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2016 Type: Article Affiliation country: Netherlands
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glycogen Storage Disease Type II / Muscle, Skeletal / Alpha-Glucosidases / Enzyme Replacement Therapy Type of study: Clinical_trials / Qualitative_research Limits: Adult / Aged / Female / Humans / Male / Middle aged Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2016 Type: Article Affiliation country: Netherlands