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City Irvine / Porto / Taipei / New York / Framingham / Durham / / Company D. Bali S.A. / L.M. Franco S.P. / Genzyme Corporation / Elsevier Inc. / Y.T. Chen D.D. / C.I. Van Capelle N.A. / CI-MPR / / Country Taiwan / United States / / / Event Product Issues / FDA Phase / Business Partnership / / Facility Blood Institute / D.R. Plant / USA Institute of Biomedical Sciences / Duke University / Pflugers Arch / Duke University Medical Center / / IndustryTerm dynamometry and quantitative magnetic resonance imaging / therapies for lysosomal diseases / therapy for infantile glycogen storage disease / loxP site / treatment of Pompe disease / therapy for Pompe disease / intracellular processing / / MedicalCondition muscular dystrophy / both infantile and adult human glycogen storage disease / lysosomal storage disorders / twitch / Muscle weakness / juvenile-onset Pompe disease / tremor / glycogen storage disease / II glycogenosis / pulmonary edema / Introduction Pompe disease / illness / Infantile-onset Pompe disease / late-onset myopathy / Pompe disease / muscle atrophy / asthma / insomnia / cardiomyopathy / disease / late-onset Pompe disease / alpha-glucosidase Acid maltase Pompe disease Glycogen storage disease / infantile glycogen storage disease / late-onset glycogen storage disease type / metabolic syndrome / muscle tremor / NIDDM / lysosomal diseases / muscle disorder glycogen storage disease / severe / infantileonset hypertrophic cardiomyopathy / / MedicalTreatment intravenous infusions / enzyme replacement therapy / adjunctive therapy / / Organization National Institute of Health / Muscular Dystrophy Association / Department of Pediatrics / National Heart / Lung / and Blood Institute / Duke University / Systemic administration / USA Institute of Biomedical Sciences / FDA / Division of Medical Genetics / European Union / University Institutional Animal Care and Use Committee / Duke University Medical Center / / Person J.C. Bruning / Gene Tex / M. Lamarca / Jian Dai / Nat / A. Van Der / Nina Raben / Van Der Beek / A.A. Wylie / Alison McVie-Wylie / J. Ward / Randy Jirtle / S.Z. Marsala / V / / Position Corresponding author at / Product albuterol / Myozyme / Strigl-Pill / rhGAA uptake / ERT / responsiveness / Lumizyme™ / / ProvinceOrState New York / North Carolina / Massachusetts / / Technology alpha / antibodies / genotyping / magnetic resonance imaging / recombination / / SocialTag