Overview of SKID Treatment - The report focuses on a clinical trial for Severe Combined Immunodeficiency (SKID), also known as "bubble boy disease," and its successful treatment [2][3] - Untreated SKID has a life expectancy of approximately 2 years [4] - A clinical trial at UCLA, led by Dr Donald Con, offered gene therapy and bone marrow transplant as potential treatments [5][6] Treatment and Success Rate - The experimental treatment involves extracting stem cells from the patient's bone marrow, adding a normal ADA gene, and reintroducing the cells after chemotherapy to rebuild the immune system [7] - The clinical trial achieved a 95% success rate, with 59 out of 62 patients experiencing completely restored immune function over a 5-year period [8] - Post-treatment, children are able to live normal lives, attending school and engaging in typical childhood activities without unusual infections [8] Impact and Future Availability - UCLA has licensed the gene therapy to a biotech company, aiming for FDA approval within the next 2 to 3 years [15] - Babies are now screened for SKID before leaving the hospital, providing parents with early awareness [13]

Medical Breakthrough - A 13-year-old patient with a rare form of leukemia, Alyssa Tapley, was facing end-of-life care after failed treatments [1][2] - The patient received an experimental gene editing treatment funded by US federal funding after other treatments failed [2][3] - The patient became the first human to try the experimental treatment [3] - The experimental treatment was successful, leading to the patient's cancer remission [4] Research & Development Impact - The successful treatment highlights the potential of gene editing research in treating rare and difficult diseases [2][4] - The case demonstrates the significant impact of research and resource allocation in medical advancements [4]