Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial.

Flanigan, Kevin M; Voit, Thomas; Rosales, Xiomara Q; Servais, Laurent; Kraus, John E; Wardell, Claire; Morgan, Allison; Dorricott, Susie; Nakielny, Joanna; Quarcoo, Naashika; Liefaard, Lia; Drury, Tom; Campion, Giles; Wright, Padraig

Flanigan, Kevin M; Voit, Thomas; Rosales, Xiomara Q; Servais, Laurent; Kraus, John E; Wardell, Claire; Morgan, Allison; Dorricott, Susie; Nakielny, Joanna; Quarcoo, Naashika; Liefaard, Lia; Drury, Tom; Campion, Giles; Wright, Padraig.

Afiliação

Flanigan KM; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, United States.
Voit T; Institut de Myologie, Université Pierre et Marie Curie Paris 6, UM 76, INSERM U 974, CNRS UMR 7215, France.
Rosales XQ; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, United States.
Servais L; Institut de Myologie, Université Pierre et Marie Curie Paris 6, UM 76, INSERM U 974, CNRS UMR 7215, France.
Kraus JE; GlaxoSmithKline, Research Triangle Park, NC, United States. Electronic address: john.e.kraus@gsk.com.
Wardell C; GlaxoSmithKline, Stockley Park, UK.
Morgan A; Prosensa Therapeutics BV, Leiden, The Netherlands.
Dorricott S; Prosensa Therapeutics BV, Leiden, The Netherlands.
Nakielny J; GlaxoSmithKline, Stockley Park, UK.
Quarcoo N; GlaxoSmithKline, Stockley Park, UK.
Liefaard L; GlaxoSmithKline, Stevenage, UK.
Drury T; GlaxoSmithKline, Stockley Park, UK.
Campion G; Prosensa Therapeutics BV, Leiden, The Netherlands.
Wright P; GlaxoSmithKline, London, UK.

Neuromuscul Disord ; 24(1): 16-24, 2014 Jan.

Article em En | MEDLINE | ID: mdl-24321374

Assuntos

Distrofia Muscular de Duchenne/tratamento farmacológico; Oligonucleotídeos/uso terapêutico; Adolescente; Biomarcadores; Criança; Método Duplo-Cego; Humanos; Mediadores da Inflamação/análise; Masculino; Distrofia Muscular de Duchenne/metabolismo; Oligonucleotídeos/administração & dosagem; Oligonucleotídeos/efeitos adversos; Oligonucleotídeos/farmacocinética

Palavras-chave

DMD; Drisapersen; Duchenne muscular dystrophy; Dystrophin; Exon 51; Non-ambulant; Oligonucleotide; Pharmacokinetics; Safety

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Oligonucleotídeos / Distrofia Muscular de Duchenne Idioma: En Ano de publicação: 2014 Tipo de documento: Article

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Oligonucleotídeos / Distrofia Muscular de Duchenne Idioma: En Ano de publicação: 2014 Tipo de documento: Article