Experimental drug hopes to slow rare muscle disease in two patients
Disease control
ENROLLING_BY_INVITATION
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…
Phase: PHASE1 • Sponsor: Cure ADSSL1 • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC