Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial.

Wagner, Kathryn R; Kuntz, Nancy L; Koenig, Erica; East, Lilly; Upadhyay, Sameer; Han, Baoguang; Shieh, Perry B

Wagner, Kathryn R; Kuntz, Nancy L; Koenig, Erica; East, Lilly; Upadhyay, Sameer; Han, Baoguang; Shieh, Perry B.

Afiliación

Wagner KR; Center for Genetic Muscle Disorders, Kennedy Krieger Institute, Baltimore, Maryland, USA.
Kuntz NL; Departments of Neurology and Neuroscience, Johns Hopkins School of Medicine, Baltimore, Maryland, USA.
Koenig E; Ann & Robert H. Lurie Children's Hospital, Chicago, Illinois, USA.
East L; Clinical Development, Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA.
Upadhyay S; Clinical Pharmacology, Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA.
Han B; Pharmacovigilance, Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA.
Shieh PB; Biostatistics, Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA.

Muscle Nerve ; 64(3): 285-292, 2021 09.

Article en En | MEDLINE | ID: mdl-34105177

Asunto(s)

Distrofina/genética; Distrofia Muscular de Duchenne/genética; Oligonucleótidos/efectos adversos; Adolescente; Niño; Método Doble Ciego; Exones; Humanos; Masculino; Mutación; Oligonucleótidos/administración & dosificación; Oligonucleótidos/farmacocinética; Adulto Joven

Palabras clave

Duchenne muscular dystrophy; RNA oligonucleotide; casimersen; exon skipping; phosphorodiamidate morpholino oligomer

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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Oligonucleótidos / Distrofina / Distrofia Muscular de Duchenne Tipo de estudio: Clinical_trials Idioma: En Revista: Muscle Nerve Año: 2021 Tipo del documento: Article

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