What treatments are on the horizon for SCN8A?
Neurocrine Biosciences received FDA approval to begin Phase II clinical trials for their drug, NBI-921352 (XEN901) for children and young adults 12-21 years old.
This drug is a selective Nav1.6 sodium channel inhibitor with potential in treating SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE) as well as other forms of epilepsy, including focal epilepsy.
What is the Purpose of the Kayak Study?
The purpose of the Kayak Study is to evaluate the efficacy, safety, and tolerability of an investigational study drug compared to a placebo (inactive drug) as add-on therapy in young people 2 to 21 years of age with SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE). The study is looking at how the study drug affects participants’ symptoms, quality of life, severity of seizures, and frequency of seizures. To see if you or your child may qualify for study participation, click here.
Praxis Precision Medicines has recently received rare pediatric disease designation for it’s compound, PRAX-562, for treatment SCN8A-DEE. See the latest community statement we helped them craft to answer YOUR questions below!