Nikolai A. Naryshkin

<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

Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( <i>SMN2</i> ) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)

Hasane Ratni, Martin Ebeling, John D. Baird et al. · Journal of Medicinal Chemistry · 2018 · 492 citations · Full text

Structural Organization of the RNA Polymerase-Promoter Open Complex

Nikolai A. Naryshkin, Andrey Revyakin, Young-Gyu Kim et al. · Cell · 2000 · 184 citations

Viral Polymerase Structure, Natural Sciences, Rna Biology +5

Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment

Daniel M. Ramos, Constantin d’Ydewalle, Vijayalakshmi Gabbeta et al. · Journal of Clinical Investigation · 2019 · 183 citations · Full text

Backgroundspinal Muscular Atrophy, Disease Progression, Pathology +23

Risdiplam distributes and increases <scp>SMN</scp> protein in both the central nervous system and peripheral organs

Agnès Poirier, Marla Weetall, Katja Heinig et al. · Pharmacology Research & Perspectives · 2018 · 160 citations · Full text