Empowered Patient Podcast

Targeting Mutations of Pediatric Genetic Epilepsies Creating New Treatment Options with Alex Nemiroff Praxis Precision Medicines

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Synopsis

Alex Nemiroff, General Counsel at Praxis Precision Medicines, focuses on developing life-altering treatments for pediatric patients with epilepsy, particularly those with genetic epilepsies known as Developmental Epileptic Encephalopathies- DEEs.  Praxis targets specific gene mutations SCN2A and SCN8A with therapies that precisely target the source of the disease. Lead drug candidate PRAX-562 is a precision small-molecule treatment that inhibits seizure-causing activity, and PRAX-222 is a type of RNA therapy that shows potential for complete disease modification. Alex explains, "There are a number of types of epilepsy. At Praxis, we focus on a few. We have a broader focal epilepsy program, and then we have a number of programs targeting these genetic epilepsies that are referred to as DEEs, Developmental Epileptic Encephalopathies. These are epilepsies that start in infancy, and they're tied most commonly to a mutation in a gene that is known to cause epilepsy. Specifically, we have at Praxis right now in th