All articles tagged with #scid
A long-term follow-up study shows that gene therapy for babies with severe combined immunodeficiency (SCID), known as 'Bubble Boy disease,' remains 95% effective in restoring immune function, with children living healthy, normal lives and the therapy nearing FDA approval.
Bella Brave, a 10-year-old TikTok star known for her health journey, passed away on July 14, 2024, after a battle with multiple severe health conditions. Her mother, Kyla Thomson, shared the news on social media, asking followers to honor Bella's legacy and bravery. Bella's story, which included numerous surgeries and hospital stays, touched millions of followers and celebrities alike.
A parent shares their experience of receiving a false positive test result for a rare genetic disorder in their newborn, highlighting the anxiety and gratitude that came with the experience. The article discusses the importance of newborn screening programs in detecting treatable genetic disorders, such as severe combined immunodeficiency (SCID), and the remarkable progress in medicine that has made these screenings highly reliable and affordable. The author emphasizes the significant impact of these advancements on infant mortality rates and expresses gratitude for the screening test that ultimately turned out to be unnecessary, recognizing the potential life-saving impact for families whose babies are identified as needing treatment.
Newborn screening for severe combined immunodeficiency (SCID) followed by early treatment has increased the five-year survival rate of children with the disorder from 73% to 87%, according to a study by the National Institute of Allergy and Infectious Diseases. The study analysed data on more than 900 children with confirmed SCID who received treatment for the condition with a transplant of blood-forming stem cells from a non-genetically matched donor at one of 34 sites in the US or Canada between 1982 and 2018. The researchers excluded infants who received stem-cell transplants from genetically matched sibling donors from the analysis, because these children had high overall survival rates throughout the study period.