Viewing 15 posts - 1 through 15 (of 25 total)
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  • #7028

    I recently heard about a company called Biomarin. they developed a “cure” to PKU called Kuvan, I think enzyme function is established by the introduction of an overwhelming amount of coenzyme that binds with the phenalanine catalyzing enzyme that is dysfunctional in us PKU folks. by binding to our dysfunctional enzyme it corrects the incorrect folding of the proteins to make our dysfunctional enzyme work!! that means that we could turn phenalanine into tyrosine and PKU is cured! please tell me anything that you know about this and about kuvan and about how to get this treatment.

    #7096
    Avatar of Sarah
    HappyMom
    Member

    To obtain Kuvan you would need to talk to your doctor at your clinic. While Kuvan can be helpful to some people it all depends on the mutation of the chromosomes. For this reason Kuvan would not benefit my son.
    Sarah

    #7097
    Avatar of Breanna
    BreaMarie91
    Member

    First of all just to clear one thing up Kuvan is not a cure. Another option for treatment maybe, but it doesn’t take PKU away. Basically Kuvan kick starts the enzyme PAH that is lacking in our bodies to help our bodies break down more phe.
    Like Sarah said, not everyone with PKU responds to Kuvan because there are so many different mutations of PKU, only some will respond. Even those who do respond might respond differently then another Kuvan responder.I know a guy who goes to my clinic and my dietitian said he was off diet, went on Kuvan and all he had to do was take meat out of his diet and his levels went from 20 to 6. Then I know a girl my age who didn’t respond at all.
    For example I’m on Kuvan and I personally think I’ve responded great to Kuvan! I went from 300 mg of phe to 1030 mg! Now I still have to drink formula, do blood tests and monitor my phe intake but of course it’s easier now because I have more freedom. Sometimes it’s hard for me to get all that phe in because the only new foods I’ve tried are regular bread, tortillas and pasta and I only like the bread. The funny thing is I’m only getting 650 mg of phe through food, the other 380 I’m adding into my formula at the end of the day with regular milk. And of course because I’m not getting all my phe in my levels can be low, except the last two levels I did came back as 8mg/dl and then 6.2mg/dl, so that just proves that even with Kuvan high levels aren’t unheard of.
    I hope this helps! nBre

    #7246
    Avatar of donna
    catcocoa
    Member

    i wanted to know if any one is takeing kuvan iam kind of
    scard to take kuvan i will be takeing kuvan this sat please let me know how anyone is responding to kuvan.

    #7630
    Avatar of Tracie
    Tracie_PKU
    Member

    Hi there,
    I don’t want to pop your bubble,but Brea is right it is not a cure.
    My Dr. thinks i will benefit from it,but still have to take a formula Phenex 2. nPlus i would have to take up to 60 pills a day. n( I am not a pill taker )LOL
    From what i’ve heard there won’t be a cure in my life time.I’m too old.LOL nMaybe Breas.
    Take care, nTracie-PKU

    #7651
    Avatar of Breanna
    BreaMarie91
    Member

    Tracie are you sure you would have to take 60 pills? That sounds a bit fishy to me because Kuvan dosages are determined by weight. I am about 100lbs and I take nine pills (900mg dosage). This is the highest dosage for somebody my size (20mg of Kuvan/kg of weight).
    I knew male adults who are obviously bigger then I am and they are on Kuvan. I’ve never heard of somebody taking more then 20 pills. Even then I personally don’t think that is too much to take because the pills are very small and I swallow all nine at once. If you don’t like to or cannot swallow pills another option is to dissolve the pills in water, apple juice and my dietitian recently mentioned Hunts Lemon pudding being safe.

    #7652
    Avatar of Tracie
    Tracie_PKU
    Member

    HI bre, nThats a great question. nMy PKU DR. believe it or not is close to 100 yrs old.,and quite honestly he makes me nervousnow.
    I love him to death i’ve had him since 1969.
    I’m wondering if i should seek out a new PKU Dr. nWhat do you think Bre?
    When i was hospitalized 4 yrs ago that is when i started on PREKUNIL tabs,and i had to take 40 of those aday.(Which Bites)
    I am not a pill taker.
    What do you recommend?
    Tracie-PKU

    #7653
    Avatar of Tracie
    Tracie_PKU
    Member

    Also Bre i should mention,
    that i have a rare mutation,and it always seems nas though i am taking entirly to many pills.
    I’m always in a PKU study in LOS ANGLES CA. nBut they don’t give me any real answers.
    And whats even better my dietition just retired.Her name is LIZ WENZ. nHave you heard of her?
    Thanks for letting me know.
    I tend to just do what they ask ,and never ask questions.
    I think that might be a problem on my part.LOL
    Take Care, nTracie-PKU

    #7654
    Avatar of Tracie
    Tracie_PKU
    Member

    Ya know whats amazing Bre nis i have always taking way over 20 ppills of anykind it seems like.
    I think i will check into it.
    I’d rather get on the coolers that you have spoken about. nThose actually sound yummy.LOL
    What do yoo think?
    Tracie-PKU

    #7665
    Avatar of Breanna
    BreaMarie91
    Member

    Tracie, Kuvan is different from PreKunil. Kuvan is just like any other medication that a person might take for conditions such as blood pressure problems, depression etc. You take Kuvan in the morning and then your done with it. They are very small, about half the size of a tylenol. nDo you see Dr Koch? I’m just assuming that’s who you see because you mentioned your from California. Talk to him about Kuvan. From what I’ve seen and heard he is a very knowledgable man. Or contact Biomarin and speak to them directly because I doubt you would need to take 60 pills if you choose to take Kuvan. Like I said I take nine pills a day, in the morning, and that is the maximum dosage for somebody my size.

    #7666
    Avatar of Tracie
    Tracie_PKU
    Member

    Hi Bre, nWhat is funny is i weigh exactly what you do.
    I also failed to mention that he has me on Avonil and NeoPhe as well.(both pills)
    Do those sound familar to you?
    I did Live in Ca. now i live in WA., and travel to LA to see him.Is that crazy or what?LOL
    That is why i feel so full off of medication instead of food.
    Let me know what you think.
    I value your opinion!
    Tracie-PKU

    #7704
    Avatar of Jennifer
    jenn_harney
    Member

    wow! your results with kuvan sound amazing bre!! but do you receive the pku newsletter?? there is a lot of controversy over kuvan right now. many feel there haven’t been enough studies to determine the long-term effects of using kuvan. virginia schuett (THE NEWSLETTER’S EDITOR!!) is against it herself. she also has a renowned doctor whose been studying pku for a long time review why he doesn’t think it should’ve been approved by the FDA. another thing about kuvan that concerns families is the price. with a median of about $75,000 per year, it’s challenging to convince an insurance provider this new product is necessary. also, once we are 25, we have to get our own insurance. there IS a maximum amount of money we are given from insurance companies in our lifetime. at $75,000 a year, i’m fearful i’d meet that quota pretty fast. so i guess it’s all a matter of personal opinion. if your willing to risk taking the medicine without knowing the longterm effects and have the money to pay for it, then go for it, but if you have your diet under control already and not a lot of extra cash, i wouldn’t take it.

    #7709
    Avatar of Breanna
    BreaMarie91
    Member

    Jenn, we do not get the National PKU Newsletters (anymore) for personal reasons. In the past my family was very involved with the PKU community because my mother was the president of the PKU Organization of Wisconsin for about eight years and we are still are. We do keep ourselves up to date with PKU related information, especially Kuvan, but we choose not to receive our information from the newsletter (It has nothing to do with Virginia herself). Although, I have read bits and pieces of the articles that you are talking about. To be honest I struggled with the decision to begin Kuvan and so did my parents, so believe me we did as much research on our own as we could and we talked in great depth with our clinic before we choose this route.
    The main reason we choose Kuvan was because as a child and into my pre-teen years I struggled A LOT with the diet. I was a completely different person then I am now and everyone who knows me will admit to that. Right before I started high school I finally started taking charge of my diet. I tried very hard to drink my formula and write down what I am, but my levels were still not where they needed to be, so when Kuvan was offered to me we thought it would be best to take Kuvan. If taking Kuvan meant that my levels would consistently be between 2-6mg/dl rather than only half of the time, then that was what we were going to do. I began Kuvan a year ago it was believed that because I have Classical PKU I would not respond, so we didn’t even think I would be on Kuvan today.
    With the long-term risks we discovered that BH4 (Kuvan) has been used for over ten years for other disorders in other parts of the world and our geneticist backed up this research. I do recognize that these other disorders are not PKU, but those who have been using BH4 are not growing extra arms and legs . I also trust my life with my PKU staff (quite literally). If they saw even the slightest issue they would have me in the clinic in two seconds. Last Christmas I was seriously considering discontinuing taking Kuvan because controversy that I heard on the Listserv that had me terrified. My doctor and dietitian came in two days before Christmas (the day after we called), during their holiday vacation, to speak with me when my mom told them how freaked out I was. In no way did they push we in either direction, they made it very clear that whether or not I stayed on Kuvan they would always support me. They were very informative and did, and still do, listen to my concerns and they helped me threw those concerns.
    Believe me insurance was a huge concern because not only do I have PKU, but I have two sisters who also have PKU and five other siblings. We estimated that without insurance my Kuvan would cost approximately $55,000/year (the average estimated cost is $57,000). Our co-pay is $50 every three months and we were guaranteed that they would continue paying for as long as I take Kuvan. Also, Biomarin has been WONDERFUL throughout my whole ‘Kuvan Journey.’ When we received our co-pay information Biomarin actually contacted our family and told us that if that even that co-pay was too much for our family they would help cover part of it, but we did not need the assistance so that was not an issue. When I am 25 I plan to use the same insurance company that my parents use. They have been using this insurance company since before I was born and even when the company was transferred to another owner they continued.
    So yes, all in all taking Kuvan might be a bit risky but…I already knew that. I knew that before I even said yes to Kuvan. I am also well aware that Virginia Schuett is completely against Kuvan, but if I listened to everyone when they were against one of my crazy ideas in life I wouldn’t get myself anywhere LOL. Besides if the day comes that, for some reason, I cannot continue Kuvan I know (and my family members would vouch for this) that I am stubborn enough and have enough will power that I could bring myself back down to 300 mg of phe.

    #7712
    Avatar of Tracie
    Tracie_PKU
    Member

    Hey Bre,
    Its Tracie,and i did speak with Dr.Koch. nHe says i will take up to 5 pills a day ,and continue my formula.(hpoefully a different kind)It tastes not the best. nHe told me i would take 2 pills in AM and 3 pills in PM does that sound right to you? nJust curious!
    I do see him 1 time a year,and do all my blood levels through the mail.
    I’m actually waiting for my blood persription in mail then i go to the closest hospital they draw it and i send it to the lab airmail. nAt least i don’t pay for none of it. nChildrens in La Ca. PKU study takes care of all my medical bills till my insurance comes through on Dec.1st.
    I don’t live in Ca.i am in washington,but seattle childrens does not support Kuvan. nSo Dr.Koch says it will be a waste of time trying to contact them. nWhich stinks.They are the closests to me and thats 7 hrs away for the clinic.(PKU)
    Isn’t that wonderful. nAnyways take care,and talk to you soon.
    Tracie-PKU

    #7713
    Avatar of Breanna
    BreaMarie91
    Member

    I was always told to take Kuvan once, in the morning, and after eating something. If Dr Koch told you that is what he wants you to do then I would assume that’s what you need to do. Maybe it’s because your on a lower dosage or something…I’m honestly not sure.

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