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Long-term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry.
Semplicini, Claudio; De Antonio, Marie; Taouagh, Nadjib; Béhin, Anthony; Bouhour, Françoise; Echaniz-Laguna, Andoni; Magot, Armelle; Nadaj-Pakleza, Aleksandra; Orlikowski, David; Sacconi, Sabrina; Salort-Campana, Emmanuelle; Solé, Guilhem; Tard, Céline; Zagnoli, Fabien; Hogrel, Jean-Yves; Hamroun, Dalil; Laforêt, Pascal.
Affiliation
  • Semplicini C; Department of Neurosciences, University of Padova, Azienda Ospedaliera di Padova, Padova, Italy.
  • De Antonio M; Centre de référence des pathologies neuromusculaires Nord-Est-Ile de France, Hôpital La Pitié-Salpêtrière, AP-HP, Paris, France.
  • Taouagh N; Centre de référence des pathologies neuromusculaires Nord-Est-Ile de France, Hôpital La Pitié-Salpêtrière, AP-HP, Paris, France.
  • Béhin A; Institut de Myologie, Hôpital La Pitié-Salpétrière, AP-HP, Paris, France.
  • Bouhour F; Centre de référence des pathologies neuromusculaires Nord-Est-Ile de France, Hôpital La Pitié-Salpêtrière, AP-HP, Paris, France.
  • Echaniz-Laguna A; Service ENMG et pathologies neuromusculaires, Hôpital Neurologique Pierre Wertheimer, Hospices Civils de Lyon, Lyon, France.
  • Magot A; Département de Neurologie, CHU Bicêtre, Le Kremlin Bicêtre, France.
  • Nadaj-Pakleza A; Centre de Référence des maladies neuromusculaires Nantes-Angers, Service des Explorations Fonctionnelles, CHU, Nantes, France.
  • Orlikowski D; Department of Neurology, University Hospital Strasbourg, Strasbourg, France.
  • Sacconi S; Pôle de ventilation à domicile, AP-HP, Hôpital Raymond Poincaré, Garches, France.
  • Salort-Campana E; CIC 1429, INSERM, AP-HP, Hôpital Raymond Poincaré, Garches, France.
  • Solé G; Centre de référence des Maladies Neuromusculaires, Hôpital Archet, Nice, France.
  • Tard C; CNRS UMR7277, INSERM U1091, IBV - Institute of Biology Valrose, UNS Université Nice Sophia Antipolis, Faculté de Médecine, Parc Valrose, Nice, France.
  • Zagnoli F; Reference Center for Neuromuscular Diseases and ALS, La Timone University Hospital, Aix-Marseille University, Marseille, France.
  • Hogrel JY; AOC (Atlantique-Occitanie-Caraïbe) Reference Center for Neuromuscular Disorders, Nerve-Muscle Unit, CHU Bordeaux (Pellegrin Hospital), University of Bordeaux, Bordeaux, France.
  • Hamroun D; CHU de Lille, Inserm U1171, Neurology Department, Reference Center for Neuromuscular Disorders, Lille, France.
  • Laforêt P; CHRU Cavale-Blanche, boulevard Tanguy-Prigent, Brest, France.
J Inherit Metab Dis ; 43(6): 1219-1231, 2020 11.
Article in En | MEDLINE | ID: mdl-32515844
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glycogen Storage Disease Type II / Alpha-Glucosidases Type of study: Clinical_trials / Observational_studies / Prognostic_studies Limits: Adolescent / Adult / Aged / Child / Female / Humans / Male / Middle aged Country/Region as subject: Europa Language: En Journal: J Inherit Metab Dis Year: 2020 Type: Article Affiliation country: Italy
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glycogen Storage Disease Type II / Alpha-Glucosidases Type of study: Clinical_trials / Observational_studies / Prognostic_studies Limits: Adolescent / Adult / Aged / Child / Female / Humans / Male / Middle aged Country/Region as subject: Europa Language: En Journal: J Inherit Metab Dis Year: 2020 Type: Article Affiliation country: Italy