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

AIMS: Risdiplam (RG7916, RO7034067) is an orally administered, centrally and peripherally distributed, survival of motor neuron 2 (SMN2) mRNA splicing modifier for the treatment of spinal muscular atrophy (SMA). The objectives of this entry‐into‐human study were to assess the safety, tolerability, p...

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Vydáno v:Br J Clin Pharmacol
Hlavní autoři: Sturm, Stefan, Günther, Andreas, Jaber, Birgit, Jordan, Paul, Al Kotbi, Nada, Parkar, Nikhat, Cleary, Yumi, Frances, Nicolas, Bergauer, Tobias, Heinig, Katja, Kletzl, Heidemarie, Marquet, Anne, Ratni, Hasane, Poirier, Agnès, Müller, Lutz, Czech, Christian, Khwaja, Omar
Médium: Artigo
Jazyk:Inglês
Vydáno: John Wiley and Sons Inc. 2018
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Original Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6303280/
https://ncbi.nlm.nih.gov/pubmed/30302786
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/bcp.13786
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