New drug enters first human tests for rare Muscle-Weakening disease
Disease control
Not yet recruiting
This is the first study in people to test the safety and side effects of a new drug called DNL952 for adults with late-onset Pompe disease, a rare genetic disorder that weakens muscles. The main goal is to see how different doses are tolerated and how the drug moves through the b…
Phase: PHASE1 • Sponsor: Denali Therapeutics Inc. • Aim: Disease control
Last updated Mar 17, 2026 12:55 UTC