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

U.S., Feb. 18 -- ClinicalTrials.gov registry received information related to the study (NCT07412821) titled 'A Phase 1b Study of Adenylosuccinic Acid (ASA-001) for Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.' on Jan. 04.

Brief Summary: 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...