What is Leigh Syndrome

  

• A rare inherited neurometabolic disorder that affects the central nervous system. 
• Begins in infants between the ages of three months and two years.  Symptoms of Leigh disease usually progress rapidly and aggressively.
• The prognosis for individuals with Leigh disease is poor. 

Learn more about Leigh Syndrome

Leigh is the Most Common Pediatric Mitochondrial Disease

   

• There is no approved therapy globally. Patients are largely reliant on nutritional supplements, physical therapy and some conventional epileptic drugs.
• By increasing mitochondrial activity, the severity of symptoms may be alleviated such as swallowing, motor function and speech.
• $1B peak orphan sales potential at supportable orphan drug pricing and unlimited peak sales potential in overall application for drug pricing.