ASH Clinical News ACN_4.14_Full Issue_web | Page 121

CLINICAL TRIALS
For adult patients with PK deficiency
who are not regularly transfused.
NOW ENROLLING
For adult patients with PK deficiency
who receive regular blood transfusions.
Do you have a patient with
PK DEFICIENCY?
Two Phase 3 studies of AG-348 are now enrolling adult patients with
pyruvate kinase (PK) deficiency.
This investigational drug is an orally available, potent, small molecule activator
of pyruvate kinase-R (PKR), the defective enzyme in PK deficiency.
For study criteria, visit
activateclinicaltrials.com
or clinicaltrials.gov.
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The safety and efficacy of AG-348 have not
been established. There is no guarantee that
AG-348 will receive health authority approval
or become commercially available for the
use being investigated.
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All rights reserved. PKD-US-0050