Adenylosuccinate synthase 1 deficient myopathy

Clinical trials for Adenylosuccinate synthase 1 deficient myopathy explained in plain language.

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Experimental drug hopes to slow rare muscle disease in two patients

Disease control By invitation only

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…

Matched conditions: ADENYLOSUCCINATE SYNTHASE 1 DEFICIENT MYOPATHY

Phase 1 • Sponsor: Cure ADSSL1 • Aim: Disease control

Last updated Jun 27, 2026 12:04 UTC

Adenylosuccinate synthase 1 deficient myopathy

Get alerted when new Adenylosuccinate synthase 1 deficient myopathy trials appear

Experimental drug hopes to slow rare muscle disease in two patients