First patients test new genetic drug for rare muscle disease
Disease control
Ongoing
This is the first human study testing ABX1100, a new drug for late-onset Pompe disease—a rare genetic disorder that weakens muscles. Researchers will check the safety of different doses in 46 healthy volunteers and patients with the disease. The study will measure how the drug mo…
Phase: EARLY_PHASE1 • Sponsor: Aro Biotherapeutics • Aim: Disease control
Last updated Mar 23, 2026 15:15 UTC