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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study.
Mercuri, Eugenio; Muntoni, Francesco; Osorio, Andrés Nascimento; Tulinius, Már; Buccella, Filippo; Morgenroth, Lauren P; Gordish-Dressman, Heather; Jiang, Joel; Trifillis, Panayiota; Zhu, Jin; Kristensen, Allan; Santos, Claudio L; Henricson, Erik K; McDonald, Craig M; Desguerre, Isabelle.
  • Mercuri E; Department of Pediatric Neurology, Catholic University, Rome, Italy.
  • Muntoni F; Centro Clinico Nemo, Policlinico Universitario A Gemelli IRCCS, Rome, Italy.
  • Osorio AN; Dubowitz Neuromuscular Centre & MRC Centre for Neuromuscular Diseases, University College London, Institute of Child Health & Great Ormond Street Hospital for Children Foundation Trust, 30 Guildford Street, London, WC1N 1EH, UK.
  • Tulinius M; NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital Trust, London, UK.
  • Buccella F; Hospital Sant Joan de Déu Unidad de Patología Neuromuscular, Universidad de Barcelona, CIBERER, ISCIII, Barcelona, Spain.
  • Morgenroth LP; Department of Pediatrics, Gothenburg University, Queen Silvia Children's Hospital, Gothenburg, Sweden.
  • Gordish-Dressman H; Parent Project Italy APS, Rome, Italy.
  • Jiang J; Therapeutic Research in Neuromuscular Disorders Solutions, Pittsburgh, PA, USA.
  • Trifillis P; Center for Genetic Medicine, Children's National Health System & the George Washington, Washington, DC, USA.
  • Zhu J; PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA.
  • Kristensen A; PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA.
  • Santos CL; PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA.
  • Henricson EK; PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA.
  • McDonald CM; PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA.
  • Desguerre I; University of California Davis School of Medicine, Davis, CA, USA.
J Comp Eff Res ; 9(5): 341-360, 2020 04.
Article en En | MEDLINE | ID: mdl-31997646
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Oxadiazoles / Distrofina / Codón sin Sentido / Distrofia Muscular de Duchenne Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Humans Idioma: En Año: 2020 Tipo del documento: Article
Texto completo
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PubMed Links
Search on Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Oxadiazoles / Distrofina / Codón sin Sentido / Distrofia Muscular de Duchenne Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Humans Idioma: En Año: 2020 Tipo del documento: Article