PK Deficiency

TIF Issues Charter of Priorities (CoP) For The Global PKD Patient Community
News

TIF Issues Charter of Priorities (CoP) For The Global PKD Patient Community

In the context of its educational and advocacy efforts within the field of other rare anaemias, beyond thalassaemia and sickle cell disease (SCD), and aiming, in particular, at the support…
Mitapivat Granted Priority Review By The FDA For Pyruvate Kinase (PK) Deficiency
Clinical News

Mitapivat Granted Priority Review By The FDA For Pyruvate Kinase (PK) Deficiency

The U.S. Food and Drug Administration (FDA) has accepted Agios’ New Drug Application (NDA) for Mitapivat for the treatment of adults with pyruvate kinase (PK) deficiency. The NDA was granted…
Agios Submits New Drug Application to FDA for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency
Clinical News

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

Agios Pharmaceuticals, Inc. announced that it has submitted a New Drug Application (NDA) for Mitapivat to the U.S. Food and Drug Administration (FDA) for the treatment of adults with pyruvate…
PK Deficiency Resources Now Available On TIF’s Website
News

PK Deficiency Resources Now Available On TIF’s Website

Recognizing the similarities of thalassaemia with other rare anaemias, particularly those affecting red blood cells, (e.g. PKD, SCD), and noting the absence of disease-specific united internationally coordinated patient organizations to…
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