Reproductive findings in male animals exposed to selective survival of motor neuron 2 (SMN2) gene splicing-modifying agents
Author:
Funder
F. Hoffmann-La Roche
Publisher
Elsevier BV
Subject
Toxicology
Reference26 articles.
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2. Oral SMN2 splicing modifiers in spinal muscular atrophy: Proof-of-mechanism and ongoing clinical studies;Marquet;Eur. J. Paediatric Neurol.: EJPN: Off. J. Eur. Paediatric Neurol. Soc.,2017
3. 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;Kletzl;Neuromuscul. Disord.,2019
4. Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA);Ratni;J. Med. Chem.,2018
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