SCN2A

Miriam's therapy for fibrodysplasia ossificans progressiva has a PDUFA date of September 26th.

Elevar Therapeutics' therapy for cholangiocarcinoma and Praxis' therapy for SCN2A and SCN8A deficiencies both have a PDUFA date of September 27th.

And on September 28th, a decision is expected on Agatis' orphan drug to treat patients with MCT8 deficiency.

And in addition to those FDA decisions, CheckRare will be attending and covering the AANEM conference focused on neuromuscular disorders, and that's starting September 29th.

I think the 14-year-old had something like a 90% reduction in seizures along with some other metrics.

But the paper also said that based off the success of these two, the relative success of these two ASOs, that it suggested that these ASOs would be useful in the broader SCN2A community worldwide.

For those that had a compatible SNP, right? But I did not understand if that meant literally those two ASOs, or you're saying they would be tweaked, the same backbone would be used, and it was the...

Yeah, go ahead.

Because...

Just by luck, there are many patients who may have the same single nucleotide variant because they happen randomly.

And SCN2A is a modest-sized gene, so there are opportunities to identify patients who have both a pathogenic mutation that causes their problem and a conserved single nucleotide variant.

If you have a conserved single nucleotide variant, the ASO will work for you irrespective of whether your toxic mutation is A, B, C, or D.