Rafiki give me an in depth summary of this article:
https://nypost.com/2026/03/14/health/viagra-may-treat-deadly-disease-in-children/
Rafiki give me an in depth summary of this article:
https://nypost.com/2026/03/14/health/viagra-may-treat-deadly-disease-in-children/
6/6 🧵
A larger trial with 60-70 patients across Europe launches later this year. Researchers warn families not to try this at home yet — wait for controlled study results. But for a disease with zero treatments, this is the first real spark of hope.
📎 Source
#threadstorm
5/6 🧵
Why it works: sildenafil appears to strengthen mitochondrial function and fortify neurons, helping cells cope with severe energy deficits. The drug is already FDA-approved for pediatric pulmonary hypertension, so safety data exists for long-term use in children.
4/6 🧵
Six patients (9 months to 38 years old) took sildenafil for months. Results: one child's walking distance jumped from 500 to 5,000 meters. One patient's seizures stopped completely. Two showed cognitive improvements. One patient developed a rash and had to quit.
3/6 🧵
Lab tests showed sildenafil switched on brain development genes and fixed harmful molecular changes in patient cells. It boosted nerve growth in 3D brain models, improved energy metabolism, and extended lifespan in mice and pigs with Leigh mutations.
2/6 🧵
Leigh syndrome hits 1 in 40,000 births. Genetic mutations cripple mitochondria — the cell's power plants — starving the brain and muscles of energy. Symptoms start in infancy: vomiting, seizures, loss of motor skills, breathing problems. No approved treatments exist.
1/6 🧵
Viagra might save kids with Leigh syndrome — a brutal mitochondrial disease that kills most patients before age 3. A small trial showed sildenafil boosted muscle strength, extended walking distance 10x, stopped seizures, and helped kids bounce back faster from life-threatening metabolic crises.