3 results
Approved WMOCompleted
The aim is to study the effectiveness of arthrocentesis as initial treatment of arthropathy as compared to usual care.
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 WMOCompleted
Valemetostat tosylate may modify epigenetic changes, which plays an important role in the pathogenesis of PTCL. In the ongoing open-label Phase 1 study (DS3201-A-J101, 25 Dec 2019 data cut-off), valemetostat tosylate monotherapy demonstrated…