I inherit a lot of adult patients that are graduating from pediatrics with classic 21 hydroxylase deficiency. And the first time I see them, we have this talk. We talk about the two things that we have to try to do. We definitely have to replace the cortisol deficiency, and then we have to try to control the disease to maintain your fertility, to prevent tumor development and so on.

But right now, what we're treating you with is not perfect, and it has problems. And so I can always give you more glucocorticoid, but I know what that's going to do. So if your disease control is not good and I know the consequences of that, the treatment that I'm give you is also not good and there are other consequences of that. So we've been kind of in a situation, well, this is good enough. This is the best we can do and we accept that and the patients accept that, or at least they reluctantly accept that.

But it's kind of like the cancer patients. Used to be just wanted to survive your cancer, but that's not good enough now. Now, we need to preserve fertility. We need to prevent the chronic complications of bone loss and diabetes and all those things that comes along with chemotherapy. And the same is now true with CAH.

I think what I would say is that normal is normal. We know from longitudinal natural history studies that have been done at the NIH and in the UK that these adults have a large burden of disease, osteoporosis, diabetes, obesity, and cardiovascular disease, cardiovascular risk factors. They still die of adrenal crises. And so we still have this problem on our hands.

And so what we want to really be able to do is to mitigate those long term complications that we can directly ascribe to glucocorticoids while we maintain their glucocorticoid replacement, stress dosing for illness, medical alert identification, and all the rest. So I think we're finally on the verge of having an ability to do that where we can look at them, look them in the eye and say, OK, now I'm going to give you this treatment because I want to make you normal. And I want you to be able to just take your glucocorticoids, which you have to do, and your fludrocortisone and have your lab tests periodically.

But you go on and live your life normally and not worry about these other complications that are going to creep up on you in the next several decades. So, I hope that it's a brighter day and a brighter window for people to start looking into where they can say, OK, I take this drug and everything's OK, rather than I take this drug and I know there's going to be problems. So that's how I look at this. I think it's freeing for the physicians and it's liberating for the patients as well.