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 - Concepedia

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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

DOI Full Paper Access

98

Citations

15

References

2018

Year

Stefan Sturm, Andreas Guenther, Birgit Jaber, Paul Jordan, Nada Al Kotbi, Nikhat Parkar, Yumi Cleary, Nicolas Frances, Tobias Bergauer, Katja Heinig,

British Journal of Clinical Pharmacology

Abstract

Risdiplam was well tolerated and proof of mechanism was demonstrated by the intended shift in SMN2 splicing towards full-length SMN2 mRNA. Based on these data, Phase 2/3 studies of risdiplam in patients with SMA are now ongoing.

References

	Year	Citations

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