Fast Track Designation for a therapy making transplants safer for children with a fatal immune disorder

Bone marrow transplant For children born with severe combined immunodeficiency (SCID) life can be very challenging. SCID means they have no functioning immune system, so even a simple infection can prove life threatening. Left untreated, children with SCID often die in the first few years of life. There are stem cell/gene therapies funded by the … Continue reading Fast Track Designation for a therapy making transplants safer for children with a fatal immune disorder