The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models - Gogliotti, Rocky G.; Cardona, Herminio; Singh, Jasbir; Bail, Sophie; Emery, Carina; Kuntz, Nancy; Jorgensen, Michael; Durens, Madel; Xia, Bing; Barlow, Courtenay; Heier, Christopher R.; Plasterer, Heather L.; et al. (Human molecular genetics, 2013)
Keywords: Administration, Oral; Animal models; Animals; Disease Models, Animal; Dose-Response Relationship, Drug; Drug Evaluation, Preclinical; Endoribonucleases - antagonists & inhibitors; Endoribonucleases - metabolism; Female; Humans; Mice; Mice, Transgenic; Motor Neurons - drug effects; Motor Neurons - pathology; Muscular Atrophy, Spinal - drug therapy; Muscular Atrophy, Spinal - physiopathology; Quinazolines - pharmacokinetics; Quinazolines - pharmacology; Quinazolines - therapeutic use; Survival of Motor Neuron 2 Protein - genetics; Survival of Motor Neuron 2 Protein - metabolism; Synapses - drug effects; Synapses - physiology