2 results
Approved WMOCompleted
CORE PERIODPrimary:* Evaluate the safety and tolerability of up to 24 weeks of AG-348 administration in patients with pyruvate kinase deficiency (PK deficiency).Secondary:* Evaluate the pharmacokinetics (PK) of AG-348 and the metabolite AGI-8702.*…
Approved WMOCompleted
Explore efficacy, safety, and feasibility (of an eventual future phase 3 trial) of intravenous immunoglobulins as treatment for patients with idiopathic inflammatory myopathies.