PK Deficiency
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News
PYRUVATE KINASE DEFICIENCY | TIF Issues Charter of Priorities For The Global PKD Patient Community
This newly launched document aspires to bring together available information about PKD in an effort to identify gaps, and to make useful imperative suggestions so as to understand what changes…
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Clinical News
MITAPIVAT | FDA Grants Priority Review To New Drug For Pyruvate Kinase Deficiency
The NDA was granted a Priority Review designation and has been given a Prescription Drug User Fee Act (PDUFA) action date of February 17, 2022, accelerating the review time from…
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Clinical News
PYRUVATE KINASE DEFICIENCY | Agios Submits New Drug Application To FDA For Mitapivat
“With this NDA filing, we are poised to deliver the first potentially disease-modifying therapy for people with PK deficiency, a chronic, lifelong haemolytic anaemia characterized by serious complications affecting multiple…
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TIF 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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