Children's Hospital Of Fudan University

This early-phase study tested a new gene-editing technique (base editing) on a patient's own blood stem cells to treat severe β-thalassemia, a genetic blood disorder requiring lifelong transfusions. Two children received the edited cells after chemotherapy, and researchers monito…

This study tested whether adding the steroid prednisolone to the usual treatment (immunoglobulin and aspirin) could reduce heart artery damage in children with Kawasaki disease. Over 3,200 children were enrolled within 10 days of fever onset. The main goal was to see if the combi…

This study looked at whether atorvastatin, a standard cholesterol medicine, is safe and can reduce skin bumps called xanthomas in children with Alagille syndrome. Fifteen children took the drug for 6 months, and doctors checked if the bumps got smaller or less severe. The goal wa…

This study tested a special probiotic pill (CBT102-A) in 15 children with phenylketonuria (PKU), a genetic condition that makes it hard to break down an amino acid called phenylalanine. The goal was to see if the probiotic could safely lower phenylalanine levels in the blood. Chi…

This study tested whether a moisturizer containing postbiotics (helpful yeast and bacteria) can lower the chance of eczema returning in children aged 0-6 years whose eczema was in remission. 98 children took part, and researchers tracked how many had flare-ups compared to those u…

This study looked at whether hydroxychloroquine, a medicine used for malaria and autoimmune conditions, can help children with a rare genetic lung disease that affects surfactant—a substance that keeps lungs open. 25 children with confirmed genetic mutations took the drug, and do…

This study tested whether playing a special multitask video game could improve attention in 40 children aged 8-12 with ADHD. The game was compared to a simpler digital grid exercise. Researchers measured attention using a computer test called TOVA and also tracked ADHD symptoms a…

This small study tested a drug called esketamine in 3 girls with Rett syndrome, a rare genetic disorder that affects development and movement. The goal was to see if weekly infusions over five weeks could improve symptoms and be safe. The trial is complete, but results are not ye…

This study followed 487 pregnant women in Shanghai to see if key nutrients like folate and vitamin D affect their baby's chance of developing eczema (atopic dermatitis) by 6 months old. Researchers measured nutrient levels in the mothers' blood and checked babies for eczema. The …

This study looked at 271 children in China with a rare lung condition called childhood interstitial lung disease (chILD). The goal was to collect information and genetic samples to find out what causes the disease. By building a database, researchers hope to improve diagnosis and…

This study looked at 346 children who needed a breathing machine (ventilator) for a long time. Researchers recorded the reasons, such as nerve or lung problems, and tracked how long children stayed on the machine and their survival. The goal was to better understand this conditio…

This study looked at 800 children who survived blood cancer to see if having a congenital heart defect raises their risk of death or other health problems later in life. Researchers also checked for shared genes between the two conditions. The goal is to better understand and man…