ProQR Therapeutics (Netherlands)

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

Nathalie Goemans, M. Tulinius, Johanna T. van den Akker et al. · New England Journal of Medicine · 2011 · 659 citations · Full text

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

Thomas Voit, Haluk Topaloğlu, Volker Straub et al. · The Lancet Neurology · 2014 · 293 citations

Placebo-controlled Phase 2, Medicine, Duchenne Muscular Dystrophy +5

Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy

Susan Mulders, Walther J. A. A. van den Broek, Thurman M. Wheeler et al. · Proceedings of the National Academy of Sciences · 2009 · 247 citations · Full text

Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect

Artur V. Cideciyan, Samuel G. Jacobson, Arlene V. Drack et al. · Nature Medicine · 2018 · 209 citations · Full text

Leber Congenital Amaurosis, Photoreceptor Cilium Defect, Ocular Disease +10

Phage display screening without repetitious selection rounds

Peter A.C. ’t Hoen, Silvana M.G. Jirka, Bradley R. ten Broeke et al. · Analytical Biochemistry · 2011 · 169 citations · Full text

Point-of-care Testing, Engineering, Phage Pool +22