Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)

计划入组 15 人 · 首次发布于 2021 年 7 月 16 日

A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency

计划入组 52 人 · 首次发布于 2015 年 6 月 22 日

A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

计划入组 27 人 · 首次发布于 2018 年 6 月 18 日

Mitapivat(Pyrukynd 5 Mg 4-week, Pyrukynd)

C₂₄H₂₆N₄O₃S

PK-101

PK-10453

Rockwell Medical

United States

Commodore Metals Corp

Amova STI ETF S$A

Agios' Mitapivat Achieves Primary Endpoint in Phase 3 Trial for Children with PK Deficiency

Agios Submits New Drug Application to FDA for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency

FDA Approves Pyrukynd as First Treatment for Pyruvate Kinase Deficiency