3 results
Approved WMORecruiting
Primary objective:To assess the puncture free survival after start of treatment with cediranib (time to first need for paracentesis or thoracentesis or time to death, which event occurred first)Secondary objectives:-To assess the palliative effects…
Approved WMORecruiting
To research if total joint arthroplasty leads to better results than trapeziectomy on the long term, with subjective and objective outcome measures.
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…