OIS Podcast | Ophthalmology's leading Podcast
Sep 8, 2026 · 9 min · 7 segments
Inherited retinal diseases can profoundly affect a child’s vision and development, yet there are no approved drug treatments for most of these conditions in children. In this episode of Eye on…
Carey PowersHostUm, Kerry, that is a really great question because most rare diseases present in children, 70% in fact, and yet 95% of all rare diseases have no treatment.
So, so there's a real imbalance in that, right? Um, if we look at where most capital investment goes, it goes to age-related diseases, diseases that present later in, uh, a person's life, and there's a few reasons for that.
They can actually respond and tell you, "Oh, yeah, I can see that," or, "I can't see that." And, you know, kids, and even in some cases we're talking toddlers and, and babies, they can't give you that same feedback.
Um, so, you know, over the years, our, um, you know, our endpoints, um, and the way that we-- the blueprint for doing these clinical trials is based on, uh, the adult population, which doesn't work.
The good news is that's changing a lot, and it's changing because of genetic testing.

... never considered before about how difficult it is to get the feedback you need in a trial-
Um, Kerry, that is a really great question because most rare diseases present in children, 70% in fact, and yet 95% of all rare diseases have no treatment.
So, so there's a real imbalance in that, right? Um, if we look at where most capital investment goes, it goes to age-related diseases, diseases that present later in, uh, a person's life, and there's a few reasons for that.
They can actually respond and tell you, "Oh, yeah, I can see that," or, "I can't see that." And, you know, kids, and even in some cases we're talking toddlers and, and babies, they can't give you that same feedback.
Um, so, you know, over the years, our, um, you know, our endpoints, um, and the way that we-- the blueprint for doing these clinical trials is based on, uh, the adult population, which doesn't work.
The good news is that's changing a lot, and it's changing because of genetic testing.

... never considered before about how difficult it is to get the feedback you need in a trial-
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