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Erythrocyte Encapsulated Thymidine Phosphorylase for the Treatment of Patients with Mitochondrial Neurogastrointestinal Encephalomyopathy: Study Protocol for a Multi-Centre, Multiple Dose, Open Label Trial.
Bax, Bridget E; Levene, Michelle; Bain, Murray D; Fairbanks, Lynette D; Filosto, Massimiliano; Kalkan Uçar, Sema; Klopstock, Thomas; Kornblum, Cornelia; Mandel, Hanna; Rahman, Shamima; Roubertie, Agathe; Scarpelli, Mauro; Sedgwick, Philip M; Baru, Moshe; Sellos-Moura, Marcia; Price, Jeanie; Horn, Patrick; Nirmalananthan, Niranjanan.
Afiliación
  • Bax BE; Molecular and Clinical Sciences, St. George's, University of London, London SW17 0RE, UK. bebax@sgul.ac.uk.
  • Levene M; Molecular and Clinical Sciences, St. George's, University of London, London SW17 0RE, UK.
  • Bain MD; Molecular and Clinical Sciences, St. George's, University of London, London SW17 0RE, UK.
  • Fairbanks LD; The Purine Research Laboratory, St Thomas' Hospital, London SE1 7EH, UK.
  • Filosto M; Centre for Neuromuscular Diseases, ASST Spedali Civili and University of Brescia, 25100 Brescia, Italy.
  • Kalkan Uçar S; Division of Inborn Error of Metabolism, Ege University Medical Faculty, 35100 Izmir, Turkey.
  • Klopstock T; Department of Neurology, Friedrich-Baur-Institute, University of Munich, Ziemssenstr. 1, 80336 Munich, Germany.
  • Kornblum C; Munich Cluster for Systems Neurology (SyNergy), Ludwig Maximilians University, Geschwister-Scholl-Platz 1, 80539 Munich, Germany.
  • Mandel H; German Center for Neurodegenerative Diseases (DZNE), Feodor-Lynen-Strasse 17, 81377 Munich, Germany.
  • Rahman S; Department of Neurology, University Hospital Bonn, 53127 Bonn, Germany.
  • Roubertie A; Galilee Medical Center, Nahariya 22100, Israel.
  • Scarpelli M; Mitochondrial Research Group, UCL London Great Ormond Street Institute of Child Health, London WC1N 1EH, UK.
  • Sedgwick PM; Metabolic Unit, Great Ormond Street Hospital NHS Foundation Trust, London WC1N 3JH, UK.
  • Baru M; Department of Pediatric Neurology, Centre Hospitalier Universitaire de Montpellier, 34295 Montpellier, France.
  • Sellos-Moura M; Institute of Neurology, University of Verona, 37126 Verona, Italy.
  • Price J; Institute for Medical and Biomedical Education, St George's, University of London, London SW17 0RE, UK.
  • Horn P; Orphan Technologies, Zürcherstrasse 19, 8640 Rapperswil, Switzerland.
  • Nirmalananthan N; Orphan Technologies, Zürcherstrasse 19, 8640 Rapperswil, Switzerland.
J Clin Med ; 8(8)2019 Jul 24.
Article en En | MEDLINE | ID: mdl-31344955

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Guideline Idioma: En Revista: J Clin Med Año: 2019 Tipo del documento: Article País de afiliación: Reino Unido

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Guideline Idioma: En Revista: J Clin Med Año: 2019 Tipo del documento: Article País de afiliación: Reino Unido