Phenylketonuria (PKU) Support Group
Phenylketonuria [PKU] is a human genetic disorder, in which the body lacks phenylalanine hydroxylase, the enzyme necessary to metabolize phenylalanine to tyrosine. Left untreated, the disorder can cause brain damage and progressive mental retardation as a result of the accumulation of phenylalanine and its breakdown products.
have any of you heard of the new treatment pegpal.. my friend is on it and she takes 2 shoots on monday and 2 on friday in the stomach she says the needle is smaller than an insulin needle and her levels went from 21 to 1 . " So I need to show you all about the new drug from bio marine I'm on, it's called pegpal and my protein levels went from a 21 to a 1..... I'm able to eat whatever I want without taking formula, and I only take 4 shots on Monday and Friday and they are smaller than an insulin needle... I do them myself being the BIG chicken that I am haha, what they did was they extracted an enzyme called phenylalanine ammonia lyase from a plant, and replaced phenylalanine ammonia lyase with phenylalanine hydroxylase which is the enzyme that completely doesn't work with classical PKU, and only sometimes works with hyper-phe. I have classical PKU and this has been working OUTSTANDINGLY and statistics show it is much better than kuvan for all types of PKU instead of a 50/50 chance, it now works for MOST people who try it! Side effects for me have only been itchyness around the injection site that I grew out of.. :) let me know if u wanna know more! It's gonna be open to the public soon!"
http://www.canpku.org/images/pdf/pegpal_explanation.pdf
just thought i would give suggestions
http://www.canpku.org/images/pdf/pegpal_explanation.pdf
just thought i would give suggestions
I was fortunate enough to find out about the clinical trial when they were looking for study subjects and ConfuzzledBrooke13, your friend must have been, as well. There is no other way to get ahold of the drug at this point. And unfortunately, they just closed the study and are no longer looking for subjects. The good news is that it should be going on the market in 2015 sometime if things continue the way they are.
I have only been on the drug since the beginning of March, and they start everyone on a very low dose to and work up to the actual maintenance dose, as there have been some who have adverse reactions. It may be interesting to note, too, that all subjects are asked not to I, myeslf, have experienced some joint pain, more in the beginning than now. There were maybe 2 days that it was really uncomfortable and now, it's more of an annoyance than anything and I'm allowed to take ibuprofen for it. However, they have told me that I'm one of the very few that has experienced this so early and to such a degree. That part of it does go away as your body gets used to the drug. Other than that, some skin reactions where I injected the drug, mostly mild, but the first time I injected in my leg the whole thigh was red and swollen. But that never happened again.
Having been on the drug for such a short period of time, we can't tell if my Phe level has been lowered yet, but I have to say, I am feeling some difference already - and I'm not even on the full dose yet. I have had bad anxiety and I'm only learning now that I've had ADHD symptoms, as well as insomnia issues, as well as depression. I can say that I have seen my anxiety level decrease, my sleep is better (I can stay asleep or at least get back to sleep when I wake), my mood is better and I just feel more focused.
I should add that all subjects were asked not to change their diet while on the study and I have not been on the PKU diet since I was 4.
I started on a tiny, tiny dose once a week for a month (barely anything in the syringe), and then I went up to about half the syringe once a week. From there, I went to the same amount 4 times weekly. Next week, I go to 7 times a week (every day) and then the week after, I'll be at the dose they assigned me for the rest of this phase of the study (I believe they are still working out what dose they will recommend once on the market, so different people are on different doses). You do have to inject yourself, but they won't let you out of their site until you and they are both confident you can draw up the drug and inject, so it's really no big deal to me any more, Sure, I still get a little ooked out, but it's gotten better over time and will continue to do so.
Anyway, I thought that I'd share my experience with the drug so far. It really is as exciting as it sounds and I can't wait to share my experiences as I go forward in this study.