Stuart W. Peltz

PTC124 targets genetic disorders caused by nonsense mutations

Ellen Welch, Elisabeth R. Barton, Jin Zhuo et al. · Nature · 2007 · 1.1K citations

Mendelian Disorder, Genetic Disorder, Genetics +4

<i>SMN2</i> splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

Nikolai A. Naryshkin, Marla Weetall, Amal Dakka et al. · Science · 2014 · 517 citations

Genetics, Molecular Biology, Therapeutics +21

The product of the yeast UPF1 gene is required for rapid turnover of mRNAs containing a premature translational termination codon.

Peter Leeds, Stuart W. Peltz, Allan Jacobson et al. · Genes & Development · 1991 · 495 citations · Full text

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Craig M. McDonald, Craig Campbell, Ricardo Erazo Torricelli et al. · The Lancet · 2017 · 446 citations · Full text

Rare Diseases, Urology, Clinical Trials +5

Ataluren treatment of patients with nonsense mutation dystrophinopathy

K. Bushby, Richard S. Finkel, Brenda Wong et al. · Muscle & Nerve · 2014 · 416 citations · Full text