Hasane Ratni

<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

Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers

Manaswini Sivaramakrishnan, Kathleen McCarthy, Sébastien Campagne et al. · Nature Communications · 2017 · 219 citations · Full text

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

Structural basis of a small molecule targeting RNA for a specific splicing correction

Sébastien Campagne, Sarah Boigner, Simon Rüdisser et al. · Nature Chemical Biology · 2019 · 148 citations · Full text

Transcriptional Regulation, Systems Biology, Medicine +13