- German researchers have found that sildenafil, the active ingredient in the erectile dysfunction drug Viagra, shows promise as a treatment for Leigh syndrome.
- A pilot study observed improvements in muscle strength, neurological, and metabolic symptoms in six patients aged nine to 38 years old over several months.
- Leigh syndrome is a rare, often fatal genetic childhood disease affecting one in 36,000 children globally, with no currently approved treatments.
- Patients in the study took low to medium doses of sildenafil, a small fraction of a typical Viagra dose, which works by increasing blood flow to treat complications like high blood pressure in the lungs.
- Researchers are now planning a Europe-wide clinical trial to further investigate the drug's efficacy for this serious hereditary condition.
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