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A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy.
Goemans, Nathalie; Mercuri, Eugenio; Belousova, Elena; Komaki, Hirofumi; Dubrovsky, Alberto; McDonald, Craig M; Kraus, John E; Lourbakos, Afrodite; Lin, Zhengning; Campion, Giles; Wang, Susanne X; Campbell, Craig.
Afiliação
  • Goemans N; Department of Pediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium. Electronic address: nathalie.goemans@uzleuven.be.
  • Mercuri E; Pediatric Neurology, Catholic University, Rome, Italy.
  • Belousova E; Research and Clinical Institute of Pediatrics, Pirogov Russian National Research Medical University, Moscow, Russia.
  • Komaki H; Department of Child Neurology, National Center of Neurology and Psychiatry, Tokyo, Japan.
  • Dubrovsky A; Fundacion Cenit, Instituto de Neurociencias, Fundación Favaloro, Buenos Aires, Argentina.
  • McDonald CM; School of Medicine, University of California, Davis, Sacramento, CA, USA.
  • Kraus JE; GlaxoSmithKline, Research Triangle Park, NC, USA.
  • Lourbakos A; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Lin Z; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Campion G; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Wang SX; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Campbell C; Paediatric Neurology, Schulich School of Medicine, Western University, London, Canada.
Neuromuscul Disord ; 28(1): 4-15, 2018 01.
Article em En | MEDLINE | ID: mdl-29203355

Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Oligonucleotídeos / Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials Limite: Adolescent / Child / Child, preschool / Humans / Male Idioma: En Revista: Neuromuscul Disord Assunto da revista: NEUROLOGIA Ano de publicação: 2018 Tipo de documento: Article

Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Oligonucleotídeos / Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials Limite: Adolescent / Child / Child, preschool / Humans / Male Idioma: En Revista: Neuromuscul Disord Assunto da revista: NEUROLOGIA Ano de publicação: 2018 Tipo de documento: Article