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

Stefan SturmAndreas GüntherBirgit JaberPaul JordanNada Al KotbiNikhat ParkarYumi ClearyNicolas FrancesTobias BergauerKatja HeinigHeidemarie KletzlAnne MarquetHasane RatniAgnès PoirierLutz MüllerChristian CzechOmar Khwaja

Published in: British journal of clinical pharmacology (2018)

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.

Keyphrases

big data
binding protein