A Phase 1b Study of Adenylosuccinic Acid (ASA-001) for Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.

The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of ASA-001 in two adults diagnosed with ADSS1 deficient myopathy. The main questions it aims to answer are: * Whether ASA-001 can be safely administered to ADSS1 deficient myopathy patients; * Whether daily treatment with ASA-001 provides benefit or slows progression of disease. Participants will: * Take ASA-001 every day for 8 months; * Visit the clinic once every 2 weeks for check-ups and tests

NCT Number	NCT07412821
Lead Sponsor	Cure ADSSL1 Collaborators: University of California, Los Angeles
Conditions	Adenylosuccinate Synthase 1 Deficient Myopathy
Enrollment	2 participants
Start Date	2026-03-26
Primary Completion	2027-12-31 (estimated)
Study Completion	2027-12-31 (estimated)
Updated on ClinicalTrials.gov	2026-04-07