Fanconi syndrome future or investigational therapies
Some of the recently introduced strategies in the management of Fanconi syndrome are provided below; of note, due to various underlying mechanisms leading to the disease, researches are on in this field https://doi.org/10.1080/21678707.2017.1259560.
- In a rare variant of Fanconi syndrome named Fanconi reno-tubular syndrome 1 (FRTS1), the patients have fatty acid oxidation problem due to a mitochondrial defect; dequalinium chloride (DECA) which s a newly introduced drug for hyperoxaluria has appeared to be effective in treatment of this syndrome by not permitting the import of unfunctional mutated protein.
- In other types of mitochondrial defects leading to Fanconi syndrome, it is of recently proposed that enhancement of this protein import by the drug sodium pyrithione can alleviate the disease.
- Consumption of different anti-oxidants has shown promising results in the treatment of Fanconi syndrome with fatty acid oxidation defects.
- It has been shown that Anti-apoptotic drugs are also very effective in Fanconi syndrome variants with cell apoptosis as a leading mechanism like tyrosinemia and cystinosis.
- Stimulation of mammalian target of rapamycin complex 1 (mTORC1), an important regulator protein in cell autophagy and lipid metabolism, by specific aminoacids or kinases is also recently suggested as a therapeutic approach for Fanconi syndrome.
- RNA silencing therapies are just recently introduced treatments targeting the down-regulation of disease genes with dominant inheritance and for instance the regulator microRNA mir21 is proposed to be investigated as a therapeutic target for some variants of Fanconi syndrome.
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