Heinrich Heine University Düsseldorf

Viagra Shows Promise as First Treatment for Fatal Childhood Disease Leigh Syndrome

- Sildenafil, the active ingredient in Viagra, demonstrated significant improvements in muscle strength and neurological symptoms in six Leigh syndrome patients aged 9 months to 38 years. - One child's walking distance increased tenfold from 500 to 5,000 meters, while another patient stopped experiencing seizures and two others showed cognitive improvements. - The European Medicines Agency has granted sildenafil orphan drug designation for Leigh syndrome, paving the way for a larger placebo-controlled trial across Europe. - This represents the first promising treatment candidate for Leigh syndrome, a rare genetic disorder affecting one in 40,000 births with no currently approved therapies.