Episode Details

Sildenafil, known as Viagra, shows promise in treating Leigh syndrome, a rare genetic disorder affecting young children. Early lab tests and animal studies indicate the drug corrects cell defects and restores normal energy production. A small trial with six patients showed improvements, including a teenager who stabilized and improved over seven years. Experts caution that results may vary due to different gene mutations and natural symptom fluctuations. These findings, published in Cell, offer hope for affected families and open avenues for further research.

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