Ontology highlight
ABSTRACT:
SUBMITTER: Alfano LN
PROVIDER: S-EPMC6617421 | biostudies-literature | 2019 Jun
REPOSITORIES: biostudies-literature
Alfano Lindsay N LN Charleston Jay S JS Connolly Anne M AM Cripe Linda L Donoghue Cas C Dracker Robert R Dworzak Johannes J Eliopoulos Helen H Frank Diane E DE Lewis Sarah S Lucas Karin K Lynch Jessie J Milici A J AJ Flynt Amy A Naughton Emily E Rodino-Klapac Louise R LR Sahenk Zarife Z Schnell Frederick J FJ Young G David GD Mendell Jerry R JR Lowes Linda P LP
Medicine 20190601 26
This analysis aims to describe the outcomes of two nonambulatory patients with Duchenne muscular dystrophy (DMD) who participated in two clinical studies. The two consecutive trials of eteplirsen (studies 201 and 202) were conducted in patients with DMD (N = 12) and confirmed genetic mutations amenable to exon 51 skipping.In study 201, 12 patients were randomized to receive once-weekly, double-blind intravenous infusions of eteplirsen 30 or 50 mg/kg or placebo for 24 weeks; patients then receive ...[more]