A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

Abstract

This 48-week, randomized, placebo-controlled phase 3 study (DMD114044; NCT01254019) evaluated efficacy and safety of subcutaneous drisapersen 6 mg/kg/week in 186 ambulant boys aged ≥5 years, with Duchenne muscular dystrophy (DMD) resulting from an exon 51 skipping amenable mutation. Drisapersen was generally well tolerated, with injection-site reactions and… (More)
DOI: 10.1016/j.nmd.2017.10.004

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@article{Goemans2017ARP, title={A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy}, author={Nathalie M. Goemans and Eugenio Mercuri and Elena D Belousova and Hirofumi Komaki and Alberto L. Dubrovsky and Craig M. McDonald and John E. Kraus and Afrodite Lourbakos and Zhengning Lin and Giles V. Campion and Susanne X. Wang and Craig Campbell}, journal={Neuromuscular disorders : NMD}, year={2017} }