New drug enters first human tests for rare Muscle-Weakening disease

NCT ID NCT07354724

Summary

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 body. The study will enroll about 32 adults who can walk at least 40 meters and have specific lung function.

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