#winning

Well, to update since I went MIA for 2 years, I've hit the jackpot.
Or that's what it feels like anyway.
As I mentioned in a previous post,
I was born with a metabolic disorder called Phenylketonuria. Or PKU for short.
(As kids, my neighbor thought PKU stood for Protein Kills U..which essentially defines PKU..so it was clever and I always try to share it.)
In November of 2013, I began participating in a study called Prism 301.
Essentially, the medicine (which is given through a pre-dosed syringe)
is called PEG-PAL. (PEGylated recombinant phenylalanine ammonia lyase or ‘PAL’)
It is an enzyme replacement for the aforementioned (in previous blog) enzyme that doesn't work properly in PKU patients.
That 'phe' that doesn't break down and covert into that healthy tyrosine for us? Yeah, Peg-Pal breaks it down.
And turns it into ammonia. And we excrete that dangerous phe through urination.
We, in turn, take Tyrosine in the form of a supplement pill.


So that's the medical side of it. Here's my results from it and why I feel as if I've won the lottery.
To manage PKU, the phe level goal is to be less than 6 mg/dL.
I don't think I've seen a level that low since middle school.
Mine stayed in the teens.
And when I started the study...29 mg/dL.
Now..as a child, when these levels aren't controlled,
short term effects include irritability, lack of concentration, lack of comprehension,
fatigue, headaches and so forth.
Long term effects include loss of brain cells, the decline of IQ points.
Not maybe. Definitely. That's what phe does to the brain.
When PKU isn't caught soon enough, like at birth, mental retardation (and I use that term medically, not as a derogatory term) is guaranteed.
As an adult, levels this high..like at 29 where mine were,
short term effects are definitely in play.
Long term? There's a good chance of dementia at an earlier age than when it normally would occur.


So, as I said, I started this drug in November.
It was a slow process, working my way up to full dosage.
This was a foreign thing entering my body and my body tried to fight it at first.
Nothing terribly bad happened...minor side effects.
My level lowered..25, 19, 9.5, 8.11 mg/dL.
And I'm currently waiting to hear of my most recent level, taken this week.
Keep in mind, to maintain this special PKU diet,
it is a strict regimen of fruits and vegetables, with a protein supplement drink to top it off.
Since November, I have been eating like YOU, reader!
Pizza, burgers, chicken, peanut butter, hot dogs! 
It's been divine!  And the future prospect of attending a cookout this summer
where I can indulge in more than just chips??? 
*girly squeal!*


This study, this Peg-Pal.  It's a miracle. It's allowing me to be "normal."
And not only that, but be normal while getting me back to healthy levels, under 6,
so I can have the most fulfilling life ahead of me.
I have God to thank for giving the people at BioMarin
the recipe of Peg-Pal for me and others like me.


This is my lottery. This is my jackpot. 
#winning




http://www.bmrn.com/pipeline/clinical-trials/pku.php