Bubble boy disease: gene therapy trial offers hope for a cure
⭐️ CURE ⭐️
Recruiting now
This study tests a gene therapy for children under 5 with X-linked severe combined immunodeficiency (SCID-X1), also known as bubble boy disease. Patients receive a low dose of chemotherapy followed by an infusion of their own blood stem cells that have been corrected with a healt…
Phase: PHASE1, PHASE2 • Sponsor: David Williams • Aim: ⭐️ CURE ⭐️
Last updated May 17, 2026 08:03 UTC