Ronnie was born on Mary 9, 2017 in California. After Ronnie and his parents were discharged from the hospital, they received a call from his physician sharing that he has tested positive for Severe Combined Immunodeficiency (SCID) in his newborn screening panel. Their physician told them to return to the hospital as soon as possible to confirm the results of the newborn screening test.
Once the family arrived at the hospital, Ronnie was admitted for confirmation testing. His test results came back positive for SCID, and thankfully, because of his early diagnosis, his physician immediately developed a treatment plan. One option would be to undergo a bone marrow transplant, with his father as a donor, to receive new, healthy cells. Ultimately, after more research and discussions with Ronnie’s physicians, his parents decided to proceed with gene therapy as his form of treatment.
The treatment was successful, and now Ronnie is happy and healthy, all thanks to the results of his newborn screening test! Watch the full video here: https://www.cirm.ca.gov/our-progress/video/stem-cell-gene-therapy-fatal-bubble-baby-disease-scid/.