PYRUVATE KINASE DEFICIENCY
Clinical trials for PYRUVATE KINASE DEFICIENCY explained in plain language.
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Lifeline extended: patients get continued access to rare disease drug
Disease control ENROLLING_BY_INVITATIONThis study provides continued access to the drug mitapivat for people with pyruvate kinase deficiency who completed earlier research studies. It's for participants who benefited from the drug but can't get it through regular commercial channels. The main goal is to monitor safety…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Phase: PHASE4 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 20, 2026 14:48 UTC
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Scientists track rare blood disorder in 500 patients worldwide
Knowledge-focused OngoingThis study is creating a long-term registry to track people with pyruvate kinase deficiency, a rare inherited anemia. It will follow up to 500 patients for 2 to 9 years to collect information on their health, treatments, and daily life. The goal is to better understand how the di…
Matched conditions: PYRUVATE KINASE DEFICIENCY
Sponsor: Agios Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:08 UTC
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Scientists hunt for hidden cause of anemia in blood cancer patients
Knowledge-focused OngoingThis study aims to find out how common a specific enzyme problem is in people with certain blood cancers and unexplained anemia. Researchers will take one blood sample from about 100 participants who have conditions like myelodysplastic syndrome (MDS) or other related disorders. …
Matched conditions: PYRUVATE KINASE DEFICIENCY
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated Mar 06, 2026 15:37 UTC