Experimental drug hopes to slow rare muscle disease in two patients
NCT ID NCT07412821
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a daily injection of ASA-001 in two adults with a rare genetic muscle disease (ADSS1 deficient myopathy). The main goals are to see if the drug is safe and if it can slow the disease. Participants will take the drug for 8 months and have clinic visits every two weeks for check-ups.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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University of California Los Angeles (UCLA)
Los Angeles, California, 90095, United States