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Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial.
Muntoni, Francesco; Guglieri, Michela; Mah, Jean K; Wagner, Kathryn R; Brandsema, John F; Butterfield, Russell J; McDonald, Craig M; Mayhew, Anna G; Palmer, Jeffrey P; Marraffino, Shannon; Charnas, Lawrence; Mercuri, Eugenio.
Afiliação
  • Muntoni F; NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, London, United Kingdom.
  • Guglieri M; The John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
  • Mah JK; Cumming School of Medicine, University of Calgary, Alberta Children's Hospital, Calgary, Alberta, Canada.
  • Wagner KR; Center for Genetic Muscle Disorders, and Departments of Neurology and Neuroscience, Kennedy Krieger Institute, Johns Hopkins School of Medicine, Baltimore, Maryland, United States of America.
  • Brandsema JF; Division of Neurology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America.
  • Butterfield RJ; University of Utah School of Medicine, Salt Lake City, Utah, United States of America.
  • McDonald CM; Lawson Health Research Institute, Children's Hospital, London, Ontario, Canada.
  • Mayhew AG; Institute of Genetic Medicine, Newcastle University, Newcastle, United Kingdom.
  • Palmer JP; Pfizer Inc, Cambridge, Massachusetts, United States of America.
  • Marraffino S; Pfizer Inc, Cambridge, Massachusetts, United States of America.
  • Charnas L; Pfizer Inc, Cambridge, Massachusetts, United States of America.
  • Mercuri E; Paediatric Neurology, Catholic University, and Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
PLoS One ; 17(8): e0272858, 2022.
Article em En | MEDLINE | ID: mdl-35998119
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article