Severe myoclonic epilepsy of infancy

And they took him over and it started our epilepsy journey.

And we went through various iterations of what this could be and couldn't be and Long story short, it ended up being a very rare epilepsy called Dravet's syndrome.

Dravet's is one of the intractable pediatric epilepsies that there's still no effective treatments or cures for.

Things have improved a little bit, but not to the point where they should be.

So upon Joe's passing in 2010, I had actually thought about trying to do something to raise money even before we lost Joe.

Right

... which targets the intractable epilepsy, um, or Dravet's.

And, uh, you know, if you... [laughs] He, he got to meet Charlotte.

He got to spend time with Charlotte.

I don't think he did.

He had something called Dravet syndrome, and that usually starts to manifest itself six, seven months.

So we didn't know anything.

Everything was normal for the first month and a half or so.

Epidiolex, approved two thousand eighteen.

Purified cannabidiol extracted from the plant for seizures, Lennox-Gastaut, Dravet syndrome, and tuberous sclerosis.

Approved for patients one year of age and older.

One year old.

They're usually in group homes or very supported at home.

They're not as responsive as, say, Dravet syndrome, which is very similar kind of cohort of patients in terms of their epilepsy severity, but their response to the diet is quite different.

Dravet is a genetic disorder with a proven only very few number of genes.

Lennox-Gastaut probably is much more polygenic.

May have something to do with that.

There's a exciting new medicine coming along.

Uh, there was just a European epilepsy congress, a drug called I zurvesnant for Dravet syndrome.

It's a, a disease that really affects small children.

They have so many epileptic seizures, it, it affects their, uh, mental development.

So we do a spinal tap, we inject the medicine, medicine goes to the brain, that's how you treat these patients.

Work for SMA, work for Dravet, work for Alexander, We got the drug to the patients for ultragenics.

We just didn't get them enough.

That's what we're saying here.

And she's taking medications.

Her disease is Dravet syndrome.

I want people to know about Dravet syndrome.

And I opened a nonprofit organization for that.

Right now, we're concentrated on helping other kids who have the same illness.

molecular biology.

And Harvey was approached by two fathers whose daughter had what's called Dravet syndrome.

And Dravet syndrome is a fairly brutal developmental encephalopathy.

Basically, these kids have a very severe form of epilepsy.

They can have 40, 50

a specific class of even onset mutation.

And that's what we kind of started the company on.

And when we built it in 2017, 2018, we had two people working for us and we were focused on just improving tRNAs and going after Dravet syndrome.