<i>SMN2</i> splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

Nikolai A. Naryshkin, Marla Weetall, Amal Dakka, +45

Science

GeneticsMolecular BiologyTherapeuticsSplicing VariantSelective Smn2 +19

Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy

Hasane Ratni, Gary M. Karp, Marla Weetall, +23

Journal of Medicinal Chemistry

Potential Novel MedicineGeneticsMolecular BiologySplicing VariantNeurology +14

Upregulation of ICAM-1 and MCP-1 but not of MIP-2 and sensorimotor deficit in response to traumatic axonal injury in rats

Mario Rancan, Vivianne I. Otto, Volkmar Hans, +5

Journal of Neuroscience Research

5-HT1A receptor agonist-mediated protection from MPTP toxicity in mouse and macaque models of Parkinson's disease

Erwan Bézard, Irene Gerlach, Rosario Moratalla, +2

Neurobiology of Disease

Macaque ModelsMedicineMptp ToxicityNeuropeptide ReceptorNeuropharmacology +6

A Synthetic Derivative of the Natural Product Rocaglaol Is a Potent Inhibitor of Cytokine-Mediated Signaling and Shows Neuroprotective Activity in Vitro and in Animal Models of Parkinson’s Disease and Traumatic Brain Injury

Thomas Fahrig, Irene Gerlach, E. Horváth

Molecular Pharmacology

Longitudinal characterization of biomarkers for spinal muscular atrophy

Štefan Holiga, Nicole Hellbach, Céline Risterucci, +14

Annals of Clinical and Translational Neurology

Sembragiline in Moderate Alzheimer’s Disease: Results of a Randomized, Double-Blind, Placebo-Controlled Phase II Trial (MAyflOwer RoAD)

Stephane Navé, Rachelle S. Doody, Merçé Boada, +18

Journal of Alzheimer s Disease

The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy

Heidemarie Kletzl, Anne Marquet, Andreas Günther, +15

Neuromuscular Disorders

Amyotrophic Lateral SclerosisGeneticsDegenerative DiseaseSpinal Muscular AtrophyNeurology +5

Effects of IONIS-HTTRx in Patients with Early Huntington’s Disease, Results of the First HTT-Lowering Drug Trial (CT.002)

Sarah J. Tabrizi, Blair R. Leavitt, Holly Kordasiewicz, +9

Neurology

Discovery of RO7185876, a Highly Potent γ-Secretase Modulator (GSM) as a Potential Treatment for Alzheimer’s Disease

Hasane Ratni, André Alker, Bjoern Bartels, +13

ACS Medicinal Chemistry Letters