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Pyruvate Kinase Deficiency - 1 Studies Found
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Nombre del estudio: A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency Condición: Pyruvate Kinase Deficiency Fecha: 2025-11-04 Intervenciones: Participants with PK deficiency were randomized to either receive AG-348, 50 or 300 mg, as initial doses, BID for the Core Period (Week 24).At t |