3 results
PrimaryTo evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with FXS as assessed by:* Incidence and severity of adverse events and serious adverse events* Changes in vital signs, laboratory assessments, and ECGs*…
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…
Primary objective: To investigate the safety of Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.Secondary objectives• To investigate the efficacy of Mim8 prophylaxis in children with haemophilia A with or without…