Treating neutropenias in two rare inborn errors of metabolism, glycogen storage disease Ib and G6PC3 deficiency

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The neutrophils are a major form of white blood cells that kill bacteria and prevent infections. When their number is abnormally decreased, patients suffer from debilitating infections. Maria Veiga da Cunha and Emile Van Schaftingen have recently understood the mechanism that causes neutropenia (too low neutrophil number) in glycogen storage disease type Ib and in G6PC3 deficiency. This knowledge led them to propose to use of an antidiabetic agent to treat these two forms of neutropenia. This treatment indeed dramatically improved the infection problems in patients. Financial support is needed to further progress in this research. References PMID: 30626647 & 32294159

— Treating neutropenias in two rare inborn errors of metabolism, glycogen storage disease Ib and G6PC3 deficiency

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donation to "Treating neutropenias in two rare inborn errors of metabolism, glycogen storage disease Ib and G6PC3..."
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Gauthier  —  2 years ago

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