New hope for Tough-to-Treat rare diseases: experimental drug targets faulty cell signals
Disease control
Recruiting now
This study is testing whether the drug cobimetinib can help control several rare disorders caused by overactive immune cells, particularly when standard treatments have failed. The trial will enroll 90 children and adults with conditions like Langerhans cell histiocytosis (LCH) a…
Phase: PHASE2 • Sponsor: Carl Allen • Aim: Disease control
Last updated Mar 17, 2026 12:55 UTC