4 results
to investigate the effect and efficacy of pyridostigmine on muscle strength and fatiquabillity in patients with SMA.
The purpose of Part A is to investigate how safe the study compound VX-152 is and how well the study compound is tolerated. The study will also investigate how quickly and to what extent the compound is absorbed into and eliminated from the body (…
The purpose of this study is to measure clinical efficacy on muscle strength and function, safety, and tolerability of NMD670 compared with placebo in ambulatory participants with Type 3 SMA
* To assess safety and tolerability of single oral doses of NMD670 in healthy male and female subjects * To assess safety and tolerability of repeated oral doses of NMD670 in healthy male subjects * To assess safety and tolerability of single oral…