Anne Marquet

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

A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a <i>SMN2</i> splicing modifier

Stefan Sturm, Andreas Guenther, Birgit Jaber et al. · British Journal of Clinical Pharmacology · 2018 · 98 citations · Full text

Longitudinal characterization of biomarkers for spinal muscular atrophy

Štefan Holiga, Nicole Hellbach, Céline Risterucci et al. · Annals of Clinical and Translational Neurology · 2017 · 48 citations · Full text

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 et al. · Neuromuscular Disorders · 2018 · 41 citations

Amyotrophic Lateral Sclerosis, Genetics, Degenerative Disease +7

The Effect of Safinamide, a Novel Drug for Parkinson's Disease, on Pressor Response to Oral Tyramine: A Randomized, Double-Blind, Clinical Trial

Anne Marquet, Katrin Kupas, Andreas Johne et al. · Clinical Pharmacology & Therapeutics · 2012 · 31 citations · Full text