PK Deficiency
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News
JUST LISTEN | Voices of PK Deficiency Episode on the Impact of New International Standards
Host Dr. Rachael Grace is joined by Tamara Schryver of Thrive with Pyruvate Kinase Deficiency and Alejandra Watson from the PKD Foundation to shed light on these ground-breaking guidelines. Listeners…
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News
SPOTLIGHT | RAIN Endorses the First International Guidelines for Pyruvate Kinase Deficiency
Pyruvate kinase deficiency (PKD) is the most common cause of chronic congenital non-spherocytic haemolytic anaemia, impacting between one in 100,000 to one in 300,000 individuals globally. Those with PKD suffer…
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TIF News
WEBINAR | Official Launch of TIF’s Nutrition Guidelines in Thalassaemia and PKD
This essential guide has been eagerly awaited by healthcare professionals and patients alike, and we are pleased to invite you to participate in the event. Date: October 13th, 2023 Time:…
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TIF News
PUBLICATIONS | Nutrition Guidelines in Thalassemia & PKD
Nutrition holds a significant position in the lives of individuals afflicted with thalassaemia and other hereditary anaemias. As part of its enduring commitment to generating guidelines and educational materials for…
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News
PK DEFICIENCY | New Episode of ‘Just Listen’ Podcast Out Now
Check out the latest episode of ‘Just Listen: Voices of PK Deficiency’ podcast, where host Dr Rachael Grace converses with Dr Matthew Heeney, a haematologist whose research is focused on the…
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News
NEW APPROVAL | PYRUKYND® Gets EU Green Light For Pyruvate Kinase (PK) Deficiency
The European Commission has granted, on November 10, 2022, marketing authorization to Agios Pharmaceuticals for PYRUKYND® intended for the treatment of PK deficiency in adult patients. People with PK…
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News
CLINICAL NEWS | PYRUKYND® Receives Positive CHMP Opinion For The Treatment of PKD in Adults
PYRUKYND® is a first-in-class, oral PK activator that was recently approved by the U.S. Food and Drug Administration (FDA). If approved by the European Commission (EC), PYRUKYND® will be the first approved disease-modifying therapy…
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News
EHA2022 | Agios Presents New Data Supporting the Benefits of PYRUKYND®In Adults With PKD
Data were presented at the European Hematology Association (EHA) Annual Congress, hosted virtually and in person in Vienna on June 9-12, 2022. “Following the FDA approval of PYRUKYND® for the treatment…
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Clinical News
PYRUVATE KINASE DEFICIENCY | FDA Approves First Drug For The Rare Inherited Anemia
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…
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News
