Nicole Risher

PTC124 targets genetic disorders caused by nonsense mutations

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

Life Sciences, Genetics, Molecular Genetics +8

<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 · 526 citations

Life Sciences, Molecular Biology, Neuroscience +17

Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical Properties

Liangxian Cao, Marla Weetall, Christopher R. Trotta et al. · Molecular Cancer Therapeutics · 2018 · 85 citations

Medicine, Oncology, Pharmacology +14

Small molecule splicing modifiers with systemic HTT-lowering activity

Anuradha Bhattacharyya, Christopher R. Trotta, Jana Narasimhan et al. · Nature Communications · 2021 · 85 citations · Full text

Life Sciences, Neuroscience, Pharmacology +17

Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy

Matthew G. Woll, Hongyan Qi, Anthony Turpoff et al. · Journal of Medicinal Chemistry · 2016 · 61 citations

Life Sciences, Medicinal Chemistry, Neuroscience +13