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Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.
Hughes, Derralynn A; Nicholls, Kathleen; Shankar, Suma P; Sunder-Plassmann, Gere; Koeller, David; Nedd, Khan; Vockley, Gerard; Hamazaki, Takashi; Lachmann, Robin; Ohashi, Toya; Olivotto, Iacopo; Sakai, Norio; Deegan, Patrick; Dimmock, David; Eyskens, François; Germain, Dominique P; Goker-Alpan, Ozlem; Hachulla, Eric; Jovanovic, Ana; Lourenco, Charles M; Narita, Ichiei; Thomas, Mark; Wilcox, William R; Bichet, Daniel G; Schiffmann, Raphael; Ludington, Elizabeth; Viereck, Christopher; Kirk, John; Yu, Julie; Johnson, Franklin; Boudes, Pol; Benjamin, Elfrida R; Lockhart, David J; Barlow, Carrolee; Skuban, Nina; Castelli, Jeffrey P; Barth, Jay; Feldt-Rasmussen, Ulla.
Affiliation
  • Hughes DA; Department of Haematology, Royal Free London NHS Foundation Trust and University College London, London, UK.
  • Nicholls K; Department of Nephrology, Royal Melbourne Hospital, Parkville, Victoria, Australia.
  • Shankar SP; Section of Vitreoretinal Surgery & Diseases, Emory University, Atlanta, Georgia, USA.
  • Sunder-Plassmann G; Division of Nephrology and Dialysis, Department of Medicine III, Medical University of Vienna, Vienna, Austria.
  • Koeller D; Doernbecher Children's Hospital, Oregon Health & Science University, Portland, Oregon, USA.
  • Nedd K; Infusion Associates, Grand Rapids, Missouri, USA.
  • Vockley G; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
  • Hamazaki T; Infusion Associates, Grand Rapids, Missouri, USA.
  • Lachmann R; Department of Pediatrics, Osaka City University Hospital, Osaka-shi, Japan.
  • Ohashi T; Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, UK.
  • Olivotto I; Jikei University Hospital, Tokyo, Japan.
  • Sakai N; Departmento Cuore e vasi, A.O.U. Careggi Firenze, Firenze, Italy.
  • Deegan P; Osaka City University Hospital, Osaka-shi, Japan.
  • Dimmock D; Lysosmal Disorders Unit, Department of Medicine, Addenbrooke's Hospital, Cambridge, UK.
  • Eyskens F; Genetics Center MS716, Children's Hospital of Wisconsin, Milwaukee, Wisconsin, USA.
  • Germain DP; Univeritair Ziekenhaus Anterpen, Edegem, Belgium.
  • Goker-Alpan O; Division of Medical Genetics, University of Versailles, Paris-Saclay University and Assistance Publique-Hôpitaux de Paris, Paris, France.
  • Hachulla E; O & O Alpan LLC, Springfield, Virginia, USA.
  • Jovanovic A; Service de Médecine, Hôpital Claude Huriez-CHRU Lille, Lille, France.
  • Lourenco CM; Department of Endocrinology and Metabolic Medicine, Salford Royal NHS Foundation Trust, Salford, UK.
  • Narita I; Hospital das Clínicas FMUSP-Ribeirão Preto, São Paulo, Ribeirão Preto, Brazil.
  • Thomas M; Niigata University Graduate School of Medicine and Dental Sciences, Niigata, Japan.
  • Wilcox WR; Royal Perth Hospital, Perth, New South Wales, Australia.
  • Bichet DG; Department of Genetics, Emory University School of Medicine, Atlanta, Georgia, USA.
  • Schiffmann R; Clinical Research Division, Hôpital du Sacré-Coeur de Montreal, University of Montreal, Montreal, Quebec, Canada.
  • Ludington E; Institute of Metabolic Disease, Baylor Research Institute, Dallas, Texas, USA.
  • Viereck C; Agility Clinical Inc., Carlsbad, California, USA.
  • Kirk J; Amicus Therapeutics Inc., Cranbury, New Jersey, USA.
  • Yu J; Amicus Therapeutics Inc., Cranbury, New Jersey, USA.
  • Johnson F; Amicus Therapeutics Inc., Cranbury, New Jersey, USA.
  • Boudes P; Amicus Therapeutics Inc., Cranbury, New Jersey, USA.
  • Benjamin ER; CymaBay Therapeutics, Inc., Newark, California, USA.
  • Lockhart DJ; Amicus Therapeutics Inc., Cranbury, New Jersey, USA.
  • Barlow C; TranscripTx, Inc., Sunnyvale, California, USA.
  • Skuban N; Parkinson's Institute and Clinical Center, Sunnyvale, California, USA.
  • Castelli JP; Amicus Therapeutics Inc., Cranbury, New Jersey, USA.
  • Barth J; Amicus Therapeutics Inc., Cranbury, New Jersey, USA.
  • Feldt-Rasmussen U; Amicus Therapeutics Inc., Cranbury, New Jersey, USA.
J Med Genet ; 54(4): 288-296, 2017 04.
Article in En | MEDLINE | ID: mdl-27834756
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Full text: 1 Database: MEDLINE Main subject: Fabry Disease / 1-Deoxynojirimycin / Alpha-Galactosidase / Molecular Chaperones Type of study: Clinical_trials Limits: Adolescent / Adult / Aged / Female / Humans / Male / Middle aged Language: En Journal: J Med Genet Year: 2017 Type: Article Affiliation country: United kingdom
Fulltext
Add to My VHL
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Full text: 1 Database: MEDLINE Main subject: Fabry Disease / 1-Deoxynojirimycin / Alpha-Galactosidase / Molecular Chaperones Type of study: Clinical_trials Limits: Adolescent / Adult / Aged / Female / Humans / Male / Middle aged Language: En Journal: J Med Genet Year: 2017 Type: Article Affiliation country: United kingdom