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Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.
McDonald, Craig M; Shieh, Perry B; Abdel-Hamid, Hoda Z; Connolly, Anne M; Ciafaloni, Emma; Wagner, Kathryn R; Goemans, Nathalie; Mercuri, Eugenio; Khan, Navid; Koenig, Erica; Malhotra, Jyoti; Zhang, Wenfei; Han, Baoguang; Mendell, Jerry R.
Afiliación
  • McDonald CM; Departments of Physical Medicine & Rehabilitation and Pediatrics, University of California Davis Health System and School of Medicine, Sacramento, CA, USA.
  • Shieh PB; Department of Neurology, University of California, Los Angeles, Los Angeles, CA, USA.
  • Abdel-Hamid HZ; UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.
  • Connolly AM; Pediatrics, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Ciafaloni E; University of Rochester Medical Center, Rochester, NY, USA.
  • Wagner KR; Center for Genetic Muscle Disorders, Kennedy Krieger Institute, Baltimore, MD, USA.
  • Goemans N; Departments of Neurology and Neuroscience, Johns Hopkins School of Medicine, Baltimore, MD, USA.
  • Mercuri E; Department of Pediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium.
  • Khan N; Pediatric Neurology, Catholic University, Rome, Italy.
  • Koenig E; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Malhotra J; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Zhang W; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Han B; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Mendell JR; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
J Neuromuscul Dis ; 8(6): 989-1001, 2021.
Article en En | MEDLINE | ID: mdl-34120909
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Distrofia Muscular de Duchenne / Morfolinos Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Adolescent / Child / Humans / Male Idioma: En Revista: J Neuromuscul Dis Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Países Bajos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Distrofia Muscular de Duchenne / Morfolinos Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Adolescent / Child / Humans / Male Idioma: En Revista: J Neuromuscul Dis Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Países Bajos