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Combination disease-modifying treatment in spinal muscular atrophy: A proposed classification.
Proud, Crystal M; Mercuri, Eugenio; Finkel, Richard S; Kirschner, Janbernd; De Vivo, Darryl C; Muntoni, Francesco; Saito, Kayoko; Tizzano, Eduardo F; Desguerre, Isabelle; Quijano-Roy, Susana; Benguerba, Kamal; Raju, Dheeraj; Faulkner, Eric; Servais, Laurent.
Afiliación
  • Proud CM; Children's Hospital of The King's Daughters, Norfolk, Virginia, USA.
  • Mercuri E; Department of Paediatric Neurology and Nemo Clinical Centre, Catholic University, Rome, Italy.
  • Finkel RS; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
  • Kirschner J; Department of Neuropediatrics and Muscle Disorders, Medical Center University of Freiburg, Faculty of Medicine, Freiburg, Germany.
  • De Vivo DC; Departments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, New York, USA.
  • Muntoni F; The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, London, UK.
  • Saito K; National Institute of Health Research, Great Ormond Street Hospital Biomedical Research Centre, London, UK.
  • Tizzano EF; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.
  • Desguerre I; Department of Clinical and Molecular Genetics, Hospital Vall d'Hebron, Barcelona, Spain.
  • Quijano-Roy S; Hôpital Necker Enfants Malades, APHP, Paris, France.
  • Benguerba K; Garches Neuromuscular Reference Center (GNMH), APHP Raymond Poincare University Hospital (UVSQ Paris Saclay), Garches, France.
  • Raju D; Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland.
  • Faulkner E; Novartis Gene Therapies, Inc, Bannockburn, Illinois, USA.
  • Servais L; Novartis Gene Therapies, Inc, Bannockburn, Illinois, USA.
Ann Clin Transl Neurol ; 10(11): 2155-2160, 2023 11.
Article en En | MEDLINE | ID: mdl-37691296
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Atrofia Muscular Espinal Límite: Humans Idioma: En Revista: Ann Clin Transl Neurol Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Base de datos: MEDLINE Asunto principal: Atrofia Muscular Espinal Límite: Humans Idioma: En Revista: Ann Clin Transl Neurol Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos