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Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.
Pena, Loren D M; Barohn, Richard J; Byrne, Barry J; Desnuelle, Claude; Goker-Alpan, Ozlem; Ladha, Shafeeq; Laforêt, Pascal; Mengel, Karl Eugen; Pestronk, Alan; Pouget, Jean; Schoser, Benedikt; Straub, Volker; Trivedi, Jaya; Van Damme, Philip; Vissing, John; Young, Peter; Kacena, Katherine; Shafi, Raheel; Thurberg, Beth L; Culm-Merdek, Kerry; van der Ploeg, Ans T.
Affiliation
  • Pena LDM; Duke University Medical Center, Durham, NC, USA. Electronic address: Loren.Pena@cchmc.org.
  • Barohn RJ; University of Kansas Medical Center, Kansas City, KS, USA.
  • Byrne BJ; University of Florida, Gainesville, FL, USA.
  • Desnuelle C; University Hospital of Nice, Côte d'Azur University, Nice, France.
  • Goker-Alpan O; O and O Alpan LLC, Fairfax, VA, USA.
  • Ladha S; Barrow Neurological Institute, Phoenix, AZ, USA.
  • Laforêt P; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie, Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux, France.
  • Mengel KE; Villa Metabolica, Centre for Pediatric and Adolescent Medicine, University Medical Center, Mainz, Germany.
  • Pestronk A; Washington University School of Medicine, St Louis, MO, USA.
  • Pouget J; CHU Timone APHM, Marseille, France.
  • Schoser B; Friedrich-Baur-Institut, Department of Neurology Klinikum München, München, Germany.
  • Straub V; Newcastle University John Walton Muscular Dystrophy Research Centre, Newcastle Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK.
  • Trivedi J; University of Texas Southwestern Medical Center, Dallas, TX, USA.
  • Van Damme P; KU Leuven (Catholic University of Leuven), Department of Neurosciences, VIB - Center for Brain & Disease Research, and University Hospitals Leuven, Department of Neurology, Leuven, Belgium.
  • Vissing J; Copenhagen Neuromuscular Center, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.
  • Young P; Department of Sleep Medicine and Neuromuscular Disorders, Universitätsklinikum Münster, Münster, Germany.
  • Kacena K; Sanofi Genzyme, Cambridge, MA, USA.
  • Shafi R; Sanofi Genzyme, Cambridge, MA, USA.
  • Thurberg BL; Sanofi Genzyme, Cambridge, MA, USA.
  • Culm-Merdek K; Sanofi Genzyme, Cambridge, MA, USA.
  • van der Ploeg AT; Erasmus Medical Center, Pompe Center, Rotterdam, The Netherlands.
Neuromuscul Disord ; 29(3): 167-186, 2019 03.
Article in En | MEDLINE | ID: mdl-30770310
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Full text: 1 Collection: 01-internacional Health context: 6_ODS3_enfermedades_notrasmisibles Database: MEDLINE Main subject: Glycogen Storage Disease Type II / Glucan 1,4-alpha-Glucosidase / Alpha-Glucosidases / Enzyme Replacement Therapy Type of study: Clinical_trials / Prognostic_studies Limits: Adult / Female / Humans / Male / Middle aged Language: En Journal: Neuromuscul Disord Year: 2019 Document type: Article
Fulltext
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Full text: 1 Collection: 01-internacional Health context: 6_ODS3_enfermedades_notrasmisibles Database: MEDLINE Main subject: Glycogen Storage Disease Type II / Glucan 1,4-alpha-Glucosidase / Alpha-Glucosidases / Enzyme Replacement Therapy Type of study: Clinical_trials / Prognostic_studies Limits: Adult / Female / Humans / Male / Middle aged Language: En Journal: Neuromuscul Disord Year: 2019 Document type: Article