Discovery of Risdiplam, a Selective Survival of Motor Neuron‑2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA) - Ratni, Hasane; Ebeling, Martin; Baird, John; Bendels, Stefanie; Bylund, Johan; Chen, Karen S; Denk, Nora; Feng, Zhihua; Green, Luke; Guerard, Melanie; Jablonski, Philippe; Jacobsen, Bjoern; et al. (Journal of medicinal chemistry, 2018)
Keywords: Chemistry, Medicinal; Life Sciences & Biomedicine; Pharmacology & Pharmacy; Science & Technology; CELLS; MICE; RESCUE; PREDICTION