Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age. Twenty-two patients were evaluated (ranging in...

City of Hope, one of the largest cancer research and treatment organizations in the United States and a leading research center for diabetes and other life-threatening illnesses, today announced that its Department of Stem Cell Biology and Regenerative Medicine received a $4.9 million grant to train the next generation of...

Reported cases of multi-system inflammatory syndrome (MIS-C) in children and adolescents who had received at least one COVID-19 vaccine dose were rare (estimated at one case per million vaccinated individuals in this age group). The reporting rate for cases without evidence of SARS-CoV-2 infection was 0.3 cases per million vaccinated...