epidermolysis bullosa

First of all, what is the condition that Ralph has?

Okay, um, so Ralph has something called epidermolysis bullosa.

Um, it's known as EB, but it's also known as butterfly skin.

So he has a form of that.

So the actual term, epidermolysis bullosa, it's sort of an umbrella.

There are multiple types, multiple subtypes.

He has the junctional type, which is, um, one of the most severe types, but under that, he has sort of an intermediate subtype.

Were they able to tell you straight away what it was, or did it take a while? Did they have to do tests and things like that?

about that.

And in that setting of recessive dystrophic EB, that again is something where you're taking, obviously you're taking mesenchymal stem cells from a totally different person.

So that's why it can have the collagen 7 mRNA in it because obviously you couldn't use the person's own that, yeah.

No, no.

So we had to form a university-based startup and look for outside money to get this stuff to go.

And so we founded a startup called Agly Therapeutics, which is now in clinical.

We've just completed a trial in recessive dystrophic EB.

We ran one on burns.

In this episode of Pedra Pearls, I welcome back Dr. Emily Gorell from the University of Colorado for a conversation about the Debra Care Conference and the momentum building across the epidermolysis bullosa community.

Dr. Gorrell reflects on the conference's remarkable growth, the renewed hope sparked by the new and emerging therapies, the importance of centering patient voices and research, clinical care, and advocacy.

Together, we explore the challenges of treatment access, the evolving needs of adults with EB, and opportunities for multidisciplinary collaboration, and why patient-focused meetings continue to be essential for driving meaningful progress.

Also, as we get started, I just wanna give a little plug for Pediatric Dermatology, volume 43, issue S2, because Dr. Gorrell was the guest editor for this issue, so be sure to check it out.

There's a link in the episode notes.

[upbeat music] Well, welcome back to the podcast, Dr. Gorrell.

I'm so excited to be talking with you again.

I guess it's been a little over a year since you joined me last to talk about the explosion in EB therapies while we were at, uh, SID back in twenty twenty-five.