3 results
• To evaluate the efficacy of JTT-251 in participants with heart failure with reduced ejection fraction (HFrEF)• To evaluate the safety and tolerability of JTT-251 following administration for 24 weeks in participants with HFrEF• To evaluate the…
Primary: To assess the efficacy of intracerebral delivery of AAVrh.10SGSH gene therapy (LYS-SAF302) in improving or stabilizing the neurodevelopmental status of MPS IIIA patients after 24 months (main cohort), compared to the expected evolution…
Main objective:• Evaluate the efficacy of IPN60130 monotherapy compared with placebo recipients in inhibiting new HO volume in adult and paediatric participants with FOP as assessed by low-dose WBCT (excluding the head)• Evaluate the safety of…