PK Deficiency

Mitapivat Receives FDA Approval For Haemolytic Anaemia From Pyruvate Kinase Deficiency
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Mitapivat Receives FDA Approval For Haemolytic Anaemia From Pyruvate Kinase Deficiency

The US Food and Drug Administration (FDA) has announced the approval of Mitapivat (PYRUKYND®) tablets as treatment for haemolytic anemia in adults with pyruvate kinase (PK) deficiency. The oral PK…
The AAC White Paper on Pyruvate Kinase Deficiency Has Just Landed on TIF’s Website
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The AAC White Paper on Pyruvate Kinase Deficiency Has Just Landed on TIF’s Website

The White Paper, based on the findings of a large international survey that looked into communication between PKD patients and caregivers and haematologists, also highlights the unmet needs and perceived…
TIF Issues Charter of Priorities (CoP) For The Global PKD Patient Community
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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
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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
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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
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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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