Can an immune drug slow a rare childhood brain disease?
Symptom relief
Completed
Ataxia-telangiectasia is a rare, inherited disease that damages the nervous system, skin, and immune system, and it has no cure. Researchers are testing sirolimus, an immune-suppressing drug, in children under 17 with the condition. The six participants take sirolimus by mouth fo…
Phase 2 • Sponsor: Sara Elva Espinosa Padilla, MD • Aim: Symptom relief
Last updated Sep 17, 2026 00:00 UTC