A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier
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Title
A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2
splicing modifier
Authors
Keywords
-
Journal
BRITISH JOURNAL OF CLINICAL PHARMACOLOGY
Volume 85, Issue 1, Pages 181-193
Publisher
Wiley
Online
2018-10-10
DOI
10.1111/bcp.13786
References
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- (2018) Hasane Ratni et al. JOURNAL OF MEDICINAL CHEMISTRY
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- (2017) C. A. Wijngaarde et al. Orphanet Journal of Rare Diseases
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- (2017) Ingrid E. C. Verhaart et al. Orphanet Journal of Rare Diseases
- Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers
- (2017) Manaswini Sivaramakrishnan et al. Nature Communications
- Drug metabolism by flavin-containing monooxygenases of human and mouse
- (2016) Ian R. Phillips et al. Expert Opinion on Drug Metabolism & Toxicology
- Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset
- (2016) Zhihua Feng et al. HUMAN MOLECULAR GENETICS
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- (2016) Manon Haché et al. JOURNAL OF CHILD NEUROLOGY
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- (2016) Robert M. Ward et al. PEDIATRIC RESEARCH
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- Disease Mechanisms and Therapeutic Approaches in Spinal Muscular Atrophy
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- (2015) Christian Czech et al. PLoS One
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- (2011) Yimin Hua et al. NATURE
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