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

NCT07412821 · clinicaltrials.gov ↗

PHASE1

Phase

ENROLLING_BY_INVITATION

Status

2

Enrollment

OTHER

Sponsor class

Conditions

Adenylosuccinate Synthase 1 Deficient Myopathy

Interventions

DRUG: adenylosuccinic acid

Sponsor

Cure ADSSL1

Collaborators

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