not kuvan, but pegpal :)

not kuvan, but pegpal :)

Avatar of Mary Caite

not kuvan, but pegpal :)

February 21, 2011 in Uncategorized

has anyone ever heard of pegpal my name is mary caite and i am 18 years old with classical pku, my life has been a struggle with keeping on diet, although it did not hinder my chances of getting into college successfully at CW Post on long island. over the summer i was wondering how im going to keep up in college and do my diet. it got my mind running every day and i was soooo nervous!!! because no matter how hard i tried, my levels were around 17-18 which is really bad : i hate to admit that i followed a poor diet because of all of these amazing stories of successful PKU students. BUT in august i was told by my nutritionist that there was a clinical trial opened to people with pku who have high levels. its a drug called pegpal and i signed up for it. It is a shot of enzymes that are injected into subcutaneous fat in your arms, legs, or stomach. (stomach is least painful believe it or not haha) but these shots are very bearable. the first couple of months were very slow because of the very small dose they gave me. it was all done just in case of any side effects. once they got to my 4th increase of the enzyme i was at a good, steady state. my levels went from an 18 down to an 8. i did not respond to kuvan, but this drug, i responded better than many. my doctors think they have a good dosage for me when i stay on diet. (which is 4 shots a week) im still being studied and going to the city once a week and donating my time and gas money to get this drug tested and passed by the FDA for it to go on market. this drug is different than kuvan which you might have heard of. both drugs are under biomarin (so it has a good reputation behind it). but kuvan only worked for 50% of people with classical pku and only worked for mainly other types. but with the pegpal, some people have shown good results and have been able to go completely off diet with classical pku! ( and that INCLUDES formula)... the enzyme in the shot replaces the defected enzyme we have. there have been better results than kuvan and in the clinic where i go to get the shots, only a couple of people did not respond as well as the rest. i HOPE this drug does well in the future because its SO GREAT to realize how CLOSE they are to finding better treatments for this condition. i only take four shots a week. this is without spreading them out. and to take shots rather than have a hard diet and annoying formula to drink is SO worth it. comment if you have any questions! id love to answer them, and if i dont know i will get back to you on friday and ask my doctor at the clinic for biomarin!!! if you have heard of it, or are currently taking pegpal, let me know! id love to see how others are doing!!!!

8 Reviews of not kuvan, but pegpal :)

  1. Registered: Jan 20, 2010

    Posts: 0

    Charlottetown, Prince Edward Island

    Wow, Thanks for sharing…It sounds hopeful.

  2. Registered: Sep 8, 2009

    Posts: 0

    , INTERNATIONAL

    Sounds amazing….BUT am from the UK and they wont even fund for Kuvan ever mind this new Pegpal, hopefully if its a success (fingers crossed) they will think about it again…Bring it on!!

  3. Registered: Dec 28, 2010

    Posts: 0

    Stockholm, INTERNATIONAL

    Wow! That´s awesome! Good for you to try this drug, it seems to help you a lot and if all goes well, it could help a lot of others like myself! The problem is that in Sweden they won´t fund for Kuvan because they think it´s too expensive so you have to be rich to afford the drug. That really sucks..

  4. Registered: Nov 12, 2009

    Posts: 0

    , Georgia

    Thank you so much for posting this! I have a 17 month old baby boy that has PKU, and your post really gives me hope for his future! I know he will be fine either way, but this drug could really make his life easier. Please keep us posted on this! Do you know when they expect this drug to be approved?

  5. Registered: Apr 13, 2010

    Posts: 0

    plant city, Florida

    i have a question, do you not need to stay on diet with the peg pal?

  6. Registered: Feb 23, 2007

    Posts: 0

    West babylon, New York

    Well, people have been able to go off diet with the pegpal BUT its different for everyone, just like kuvan, because its still a clinical trial, and many tests are being done to make sure its safe, it takes a while to get up to that point of being able to go off diet, for me, i have been responding well to the drug but i have not been able to go off diet yet :) but hopefully i can soon!

  7. Registered: Jun 8, 2008

    Posts: 0

    , INTERNATIONAL

    Tanks for sharing your story!! It also gives me hope for the future of my daughter.

    I was wondering how many phe or protein you ate before you begon with Peg Pal?

    In your story you said that not everybody is responding ass well as the rest, but is everybody responding?
     

  8. Registered: Sep 28, 2016

    Posts: 0

    No Location

    Hi. Ive been on pegpal since Sep2015 & just reached level of 3.2 (lowest since birth). For study reasons, ive been asked to watch my protein intake & keep it at a certain steady range (so they can calculate effects from a certain point). Other than journaling for 3days monthly what im eating & amounts (dietitian calculates how much im eating), i am not restricted at all in diet. I was a partial responder to kuvan, but that didnt help anymore than self-dieting w/o mgmt help.

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