3 results
Approved WMOCompleted
The purpose of the study is to assess the efficacy and safety in adolescents of AFQ056 in treating behavioral symptoms characteristic of patients with FXS. Data from this study will be used to support the registration of AFQ056 in this population…
Approved WMOCompleted
To evaluate the safety, tolerability, PK, PD and efficacy of intravenous (IV) ATB200 alone and when co-administered with oral AT2221.
Approved WMORecruiting
To assess the safety and tolerability of PLN 74809 in participants with PSC and suspected liver fibrosis