3 results
Approved WMORecruiting
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…
Approved WMOWill not start
• 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…
Approved WMORecruiting
The objective is to determine whether etidronate can halt or attenuate disease progression in patients with Fahr*s disease or syndrome.