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Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

DOIFull Paper Access

659

Citations

23

References

2011

Year

Nathalie Goemans, M. Tulinius, Johanna T. van den Akker, Brigitte E. Burm, Peter F. Ekhart, Niki Heuvelmans, Tjadine M. Holling, Anneke A. M. Janson, Gerard Platenburg, Jessica A. Sipkens,
New England Journal of Medicine

Abstract

Systemically administered PRO051 showed dose-dependent molecular efficacy in patients with Duchenne's muscular dystrophy, with a modest improvement in the 6-minute walk test after 12 weeks of extended treatment. (Funded by Prosensa Therapeutics; Netherlands National Trial Register number, NTR1241.).

References

YearCitations

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