Bubble boy disease gene therapy shows promise in early trial
Disease control
Recruiting now
This study tests a gene therapy for children with SCID-X1, a severe immune disorder often called 'bubble boy disease.' Patients receive their own blood stem cells modified with a healthy gene, plus a low dose of chemotherapy to help the cells take hold. The goal is to restore imm…
Phase: PHASE1, PHASE2 • Sponsor: David Williams • Aim: Disease control
Last updated May 02, 2026 12:42 UTC