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HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH REDUCED TOXICITY CONDITIONING REGIMEN IN MITOCHONDRIAL NEUROGASTROINTESTINAL ENCEPHALOPATHY SYNDROME (MNGIE)

2023-01-10

Abstract excerpt

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a rare autosomal recessive disorder due to mutations in the TYMP gene. Clinical findings are characterized by neurologic manifestations and severe gastrointestinal dysfunction. The syndrome is usually fatal, the most effective treatment appears to be hematopoietic stem cell transplantation (HSCT). <h4>Aim:</h4> In this retrospective study, we evalua...

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Literature Corpus work
f81d4348-6658-56f6-b39d-b863def49ecb
DOI
10.22541/au.167335388.81134587/v1
Open publication

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HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH REDUCED TOXICITY CONDITIONING REGIMEN IN MITOCHONDRIAL NEUROGASTROINTESTINAL ENCEPHALOPATHY SYNDROME (MNGIE)DOI 10.22541/au.167335388.81134587/v1
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