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Spring Survey 2012

Looking Ahead

Program: Living with PKU: Patient Approaches to the Management of PKU
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Andrew Schorr:

I love that lady, and she offers a great deal of hope. Debra Hook, are we in a new era of the management of PKU with the addition of Kuvan and other promising research?

Ms. Hook:

I think so because I think it has given us an option, and I think it’s pushed and made insurance companies and others aware that there is not going to be one treatment, that you will be on multiple treatments or what we call adjunct treatments. For some people they are able to go off diet and just be on Kuvan like John. There are others who will have to do multiple therapies, and formula has really changed. We really haven’t talked about that but there are different forms of it; there’s bars, there’s pills, there’s low volume, there’s modules and I think really we are getting to the point where we are telling insurance companies and those who are on public forms of health insurance that this is the norm. This is where we want to be, and you get more compliance, people have richer and fuller lives by having multiple therapies, and I think that the thought of something else on the horizon I think really is going to change how we view and how we treat PKU in general.

Andrew Schorr:

So John you’ve been living it, John Jr. John Sr. has too in living with it, but John Jr. so what would you say if we have teenagers listening, and we know Mary is with us from Pennsylvania and I’m sure others and parents, to just look forward to a full life in that while you were a very full responder to Kuvan, somebody else may try it, and it may be different for them, or it may be a mix of diet, formula or whatever comes down the pike as far as feeling that they can have a full life and to keep pushing in that direction?

John Adams, Jr.:

Well I think for any PKU patient whether you are on a special access drug or not you ought to put the impetus on yourself to monitor your diet. It’s really important; especially important PKU patients to know what they are putting in their body and to take responsibility for that. You know you are going to go through your trials, and you may veer off diet, but as you get older you start to figure out that it’s very vital that you really pay attention to your nutrition and that’s what I would say. Do your research, educate yourself as much as you can, know what you are putting in your body, and you life will be better for it.

Andrew Schorr:

Amen, and let’s talk to your dad. Dad you’ve worked hard to let people know they are not alone and so questions are going to come up. There are many that we couldn’t answer today, but they still have a place to turn with the listservs and other resources. BioMarin is providing resources as well, and great providers like Debra, right?

John Adams:

Absolutely, and one of the great things is that there is a new actually Genetic Metabolic Dieticians, International’s professional association for example. They have a website. There is information available there. We have a Canadian PKU and Allied Disorders Association. We’re new but we’re growing rapidly, and I am very proud of our website at www.canpku.org.

Andrew Schorr:

Okay, John Adams, Sr. downstairs in the lobby of the hotel in San Diego escorting your son, thank you so much and thanks for the work you do with rare disorders and in support in North America with PKU. John Jr. up there in the hotel probably lying on the bed, ready to go to the beach, thank you for sharing your story with us too John.

John Adams, Jr.:

Thank you for having me.

Andrew Schorr:

Okay, and Debra Hook from University of California Irvine, and I know you go around in the area and help people with these metabolic disorders, thank you for your dedication to doing it too. It sounds like we’ve got a great story coming, developing further and certainly for people who learn about a new drug like Kuvan, wonder if they can talk to you about it, they can find out about the other options, and as we mentioned in our second program there is support. There is financial support both in the drug company, the medical food people through the National Association of Rare Disorders. They’ve been helping too, so places to turn. Debra thank you for what you do.

Ms. Hook:

Thank you so much.

Andrew Schorr:

Just to wrap up then, remember we have done two previous programs; the first one heavy duty on treatment. That was our May 8th program. The replay and transcript is there for you on www.patientpower.info in the special edition section. Then we talked about access to care, all the insurance and reimbursement issues. That’s there and now this one. We’ll add the replay for this program probably tomorrow and then add the transcript as quick as we can. So it’s all there for you. Tell a friend, and I hope we’ll get to do more, but I want to thank you for participating in our PKU series. I like to say knowledge can be the best medicine of all. I’m Andrew Schorr wishing all our friends in the PKU community all the best.

Please remember the opinions expressed on Patient Power are not necessarily the views of BioMarin, our partners, or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

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