Article

Calcitriol treatment is safe and increases frataxin levels in Friedreich Ataxia patients

2023-10-30

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background</h4> Calcitriol, the active form of vitamin D (also known as 1,25-dihydroxycholecalciferol), improves the phenotype and increases frataxin levels in cell models of Friedreich Ataxia (FRDA). <h4>Methods</h4> Based on these results, we started a pilot clinical trial in which a dose of 0.25mcg/24h was administered to 15 FRDA patients for a year. Evaluations of neurological function ch...

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Literature Corpus work: 98b285d8-7b26-5523-85d4-895c97263a0c
DOI: 10.1101/2023.10.28.23297713

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