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The NDA is supported by data from a double-blind, crossover phase 3 study evaluating SL1009 in young children with a confirmed pathological mutation in the pyruvate dehydrogenase complex.
Pyruvate kinase deficiency is the most frequent abnormality of the glycolytic pathway and, together with a deficiency in glucose-6-phosphate dehydrogenase (G6PD), ...
Pyruvate dehydrogenase E1-alpha deficiency (PDHAD) results in lactic acidosis and hyperpyruvatemia. Two patients with PDHAD, a man with a p.R263Q mutation, and a girl with a p.C145del mutation in ...
All 3 patients with the pyruvate kinase deficiency were found to have elevated values for erythrocyte glucose-6-phosphate dehydrogenase, phosphoglycerate kinase and acid phosphatase.
PYRUVATE DEHYDROGENASE COMPLEX . Indications: Pyruvate dehydrogenase complex deficiency, typically associated with post-prandial lactic acidemia with a normal lactate/pyruvate ratio, can be assayed in ...
A lactate/pyruvate ratio less than 25 suggests pyruvate dehydrogenase deficiency (PDH) or other disorders of pyruvate metabolism, whereas a ratio greater than 25 suggests a respiratory chain defect.
Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA ...
Just before she turned one, Harlow was diagnosed with Pyruvate Dehydrogenase Complex Deficiency, commonly called PDCD. In children with PDCD, there is a problem with one of the primary steps that ...
ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, Jan. 28, 2025 /PRNewswire/ -- Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, announced today that the U.S. Food and Drug ...
ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, Jan. 28, 2025 /PRNewswire/ -- Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, announced today that the U.S. Food and Drug ...