Thursday, October 21, 2010

Day 159...The Google ban has lifted!



It's been about a month since we learned our wonderful (almost 1 year old!) Rhys is an "angel." Since then, I've been studying and reading everything on the internet about Angelman Syndrome or AS.  (Rob has lifted the Google ban…I think.)

It wasn't long before my trusty Google Search brought me to the marvel of a man named Dr. Weeber, who along with his colleges at Vanderbilt, have successfully reversed the neurological deficits in mice infected with AS. 

The more I read the more hope I have.  He remains very focused on the CamK2 molecule as a very significant one in the brain. This team is now trying to turn on the UBE3A gene, which is a protein as well. To activate, or turn-on that gene, they are injecting viral vectors so that it can start working again. Obtaining control of this UBE3A gene is key to successfully finding a cure for AS.  (Cousin Paul…can you translate to layman’s terms for me over Thanksgiving dinner?)

Interestingly, UBE3A also plays a big role in other more prevalent conditions like Rett Syndrome and autism. 80% of all autistic children have a gene duplication of this gene, while Rett Syndrome patients tend to be missing it. 


While Dr. Weeber is often approached by pharmaceutical firms who want to partner with him, he benefits by remaining autonomous. These firms are very motivated to find a "treatment" for AS finding a "cure" is not quite as lucrative for them. For that reason, Dr. Weeber uses government grants, and has benefited greatly from the administrations stimulus package. He is an honorable man with honorable intentions to find us a cure!


His research shows that the cause of lack of speech in AS patients is due to the fact that the imprinting is happening deep in the brain's speech centers. In hopes of correcting this symptom of AS, there is a clinical trial starting now in Boston which will explore the use of a drug called Levadopa, as a way to stimulate speech. This drug is typically used to improve muscle tone in Parkinson's patients.


So the question most on my mind... does he really believes a cure is possible for those like my sweet Rhys, who is already 1?


Of course! He explains it like this: Rett Syndrome produces a protein that effects how numerous other proteins in the body are made. In all, there are 30,000 proteins out there (who knew?). So, in the case of Rett's, it is a very extensive process to address each and every one. The great news for us, is that scientists already know the genetic cause for AS and it is identified in only one molecule - CamK2. So, when he can fix that one molecule, he will truly be able to offer us the miracle of a cure. Yippee!!


We are thrilled about his reversal success with mice, but what about humans? How soon can we expect clinical trials on people? More good news here. Dr. Weeber is starting to pursue pharmacological substances which are already FDA approved. What does this mean? It means that once he finds the right mix, he can go right into clinical trials and use people, and he doesn't think it is too far out.


So why am I rambling and wasting your perfectly precious time?  First, because I know many of you (like me) are still struggling to fully understand this syndrome that little Rhyser has.  Second, because it takes a village to raise a child and a village to raise awareness of AS.  The more we know the more we can help, right?


I feel elated when I read the progress that is being made towards a solution for AS. We couldn't have a nicer, more dedicated man in our corner.   I am so excited, because Dr. Weeber will be the keynote speaker at the
2011 ASF Biennial Conference in Salt Lake City in July 2011.  You can bet I will be there for that.

Thanks for letting me ramble.  It helps me to pass information along.  It helps me to know that someone is doing something to help these precious kids.  It gives our family hope…

On a side note, Rhys has had his helmet for a week now.  (For his flat head, completely unrelated to Angleman’s…he is just lucky!)  The good news is that he seems to be getting used to it, and it has saved the noggin in several Bodie related accidents!


Wednesday, October 13, 2010

Day 150...Pumpkin Patch

We decided to welcome the crisp autumn weather with a nice family trip to the pumpkin patch.  After some u-turns and a nice drive in the countryside we found Linder Farms.  Papa and Nanny joined in on the fun too!  So off we went on the hayride!

It was hard work picking just the right pumpkins.  McCall and Bodie immediately picked a little one for Rhys, but their pumpkin choices were much  harder.  After much deliberation they decided and off we went with 5 perfectly imperfect pumpkins.
Then we ventured into the petting zoo...and unfortunately it solidified in Rob that he desperately wants a goat.  Ya, that is just what we need.  Along with the goats there was a camel, pigs, a pony, sheep and a KANGAROO!  It was so cute...I kinda want one of those (just kidding)!



Then we snacked on the delicious donuts that I have heard so much about...all of the rave reviews did not steer us wrong.  The mini donuts did not disappoint.  I think I felt my arteries slowly clogging with deep fried sugary goodness.  It was getting really dark and cold, so the kids jumped in the corn box and made a "corn angel".  Weird, I know...


 The pumpkin picking took so long, that we didn't have time to get through the corn maze.  I still have the tokens, so we will make it back there someday.  Darn, I might have to pick me up a few more mini donuts...yummmmm!

 

Sunday, October 10, 2010

Day 148...Happy 7th Anniversary!

Wow, I cannot believe it has been 7 years.  We have had quite a roller coaster of a marriage.  Lots and lots of ups, and a couple downs, but I am so glad that Rob and I get to share these together.  I am truly lucky to be married to my best friend.  We have done a lot in seven years...

I can't wait for the next seven, and the seven after that and so on.  I hope we make it to 60 years of wedded bliss like my Papa & Grandma Rene!  I love you Babe!

Day 147...Tailgate party

We attempted yet another BSU tailgate party.  This time several factors contributed to its sucess.  For example, it was 70 degrees, not 100 degrees; I brought my wallet so I did not have to park 2.5 miles away and push a stroller of three.  Anyways, it was almost fun.  (I don't consider being outnumbered 3 to 1 in a very public place to be on my list of the most fun ever!) 

We had fun at the Smith tailgate and then checked in with a few friends too.  I forgot to pull the camera out until the stroll back to the car, by which time Bodie was in a, shall we say, FOUL mood. 
 

The Broncos won, we partied and the kids went to bed quick and quiet!  I consider that a success!

Monday, October 4, 2010

Day 142...Monkey Bizness

Rhys had another EEG this morning to get a good look at the amount and types of seizures the epilepsy is producing.  He wasn't too awfully bothered, just a lot of wires to pull!  He had to stay hooked up a little longer because he didn't have any seizures during the first portion of the test...weird that we wanted him to have episodes.



After we got out of that crazy hospital, we picked up sissy from school and went to Monkey Bizness.  The kids had tons of fun...so did Daddy!

Friday, October 1, 2010

Day 139...Two times in one day???

I couldn't resist.  We had a great day.  I needed a day off.  We did laundry, more laundry and even more laundry.  Not exactly how I wanted to spend my day off, but oh well.  Bodie and McCall played outside, instead of having naps.  Bodie then turned the hose on himself...weirdo.  I stripped him down, and we came inside.  I went in search of some clean clothes for him (we had a lot, hence the laundry) but when I came back in the room, less than a minute later....


sound asleep.  Naked as a J-Bird.  (That is the weirdest saying...I have never seen any type of bird wear clothes.)

Day 139...the Vian "men"

It was an early morning around our house, so before work Daddy stopped to relax for a minute with the boys. 
They are so cute.  Rhys looks just like Bodie, who looks just like Daddy.  I guess there is no question to who their father is?  (Not that there was a question.  I don't belong on the Maury show!)

It is nice to relax after another stressful day.  Rhys has continued to have "absence seizures".   
Absence seizures — also known as petit mal seizures — involves a brief, sudden lapse of conscious activity. They involve a slight twitch of his mouth and sometimes a small bi-lateral arm jerk.  It looks like he is merely staring into space for a few seconds and then its over.  None the less, it still scares the shit out of me.  So after a quick trip to the pediatric neurologist, and 5 of these episodes in an hour and a half, we now have him on an anti-convalescent medication.  Rob and I were very reluctant to give him medication, but it is truly necessary to make sure his development and fabulous progress is not affected.  So more grey hair for me...