The Children's Hospital Of Zhejiang University School Of Medicine

This study is testing whether adding a drug called selinexor to standard chemotherapy works better for children with a specific, high-risk type of acute myeloid leukemia (AML) linked to a gene called NUP98. The goal is to see if this combination helps more children achieve remiss…

This early-stage study is testing the safety and initial effectiveness of a therapy called CAR-T for people with severe, treatment-resistant lupus. The therapy involves modifying a patient's own immune cells to target and remove harmful B cells that drive the disease. The goal is…

This is an early-stage study to see if a new gene therapy called AAV9.hMCOLN1co is safe for children with Mucolipidosis Type IV (MLIV), a rare and serious genetic disorder. The therapy aims to deliver a working copy of the faulty gene to try to control the disease. The study will…