Category: News

My test results are in. I have to undergo a battery of tests every 6 months. So I had a complete bloodwork done, chest x-ray, 2D Echo, ECG, Urinalysis and a Hearing test. Whew.. That was a lot.

I was a bit worried at first but after showing it to my doctor she eased my fears by pointing out that some of the results were expected but not worse than before. It actually got better.

The most notable of the test results were my CPK MB levels. With Pompe patients this marker is high. A high level of CPK MB means there’s muscle damage. When I started ERT my levels were 10 points higher than normal. On my third month it was only 2 points higher and now on my 6th month of treatment, they’re normal already. Hooray!

As for the others, my creatinine is low, magnesium is a tad high and the SGOT level is high. The low creatinine is nothing to worry about my doctor said. It’s probably because I’m under weight. It would be bad if it was high. As for the magnesium it’s just a tad high so nothing really to be bothered about. As for the SGOT, although it’s high, it’s lower than the previous one. So there’s progress

Chest x-ray is almost the same although she said that I should go see the pulmonologist just to be sure. Same with the 2D echo and ECG, we will consult the cardio.

So far things are looking ok. I do need to put on weight though… I really do.

I just updated the “Fight Pompe Cafepress Store”:http://www.cafepress.com/fightpompe. Some new things to note.

1. *Premium Store* I upgraded the store to a premium store. The reason being is that with a basic store you can only sell one design per item. Ex. you can only make on design of the Ringer T shirt. You can’t add new designs to that particular shirt. The work around to this would be to open another basic store for a new design set. I didn’t want to do that. With a premium store you can make unlimited designs for a particular product.

It was also a good thing that Cafepress decided to offered to comp the first 6 months of the premium store. Which means it’s free for the first 6 months. By that time, I hopefully should have raised enough funds to pay for the monthly payments.

!/images/17.jpg (Families Unite to Fight Pompe T-shirts)!

2. *New Designs* I added a new shirt design. If you’ve read my post on the “2nd Fight Pompe Illustration”:http://www.fightpompe.com/article/43/2nd-fight-pompe-illustration I incorporated this to some shirts. Although I did modify the design a little. Watch the “Fight Pompe Store”:http://www.cafepress.com/fightpompe and this blog to for new products. I’ll be adding them in the next few weeks.

Hopefully these new changes wil mean good things for Fight Pompe. With Myozyme coming out in the market the Pompe community should now be really creative in looking for ways to make sure that every single patient gets Myozyme regardless of financial situation. Hopefully Fight Pompe can help even in a small way.

April 28, 2006 is a very special day for the Pompe Community. The FDA approved a biologics license application for Myozyme. Bringing to reality that a treatment for Pompe disease is now available for patients.

This is truly a day worth remembering. It’s a big thing for Pompe patients like me. It’s a fulfillment of a life long dream to see a treatment for this dreaded disease. It now gives patients something to look forward to.

While the approval of the FDA is great news, this is just the beginning. We’ll have to see how soon and if all patients will have access to Myozyme. It’s going to be expensive. I just hope that Genzyme, government agencies, patient organizations, other non-profit organizations and patients themselves all work together to make sure that no one suffering from Pompe disease doesn’t have access to Myozyme just because he/she can’t afford it. One of the greatest injustices in the world is people not having access to medicine just because they don’t have money. The treatment is there and the stumbling block is money. It shouldn’t be that way…

Enough of that, it’s a great day!!! Congratulations to Genzyme for getting the product to market. Many thanks to the FDA for approving Myozyme for the broad approval. Thanks to the patient organizations such as “AMDA”:http://www.amda-pompe.org for the tireless effort in pushing for a treatment. Lastly, good luck to all Pompe patients and their families, this is the start of a new life for all of us. Let’s hope it goes well…

Genzyme’s press release can be found “here”:http://www.genzyme.com/corp/investors/GENZ%20PR-042806.asp and the FDA’s press release can be found “here”:http://www.fda.gov/bbs/topics/NEWS/2006/NEW01365.html

Now as Kool & The Gang sang…

bq. Celebrate good times, come on! (Let’s celebrate)
Celebrate good times, come on!
It’s a celebration!
Celebrate good times, come on! (Let’s celebrate)

So it’s been awhile since I’ve been able to post an entry. It’s been a busy time for me and the rest of the community/world as well. I thought I’d post some interesting news from the medical community.

h3.European Commission Approves Genzyme’s Myozyme

A pivotal moment in Pompe history, Myozyme has been given approval by the European Commission as a long term treatment for Pompe Disease. Myozyme is an Enzyme Replacement Therapy (ERT) that aims to treat people suffering from Pompe Disease. It’s not a cure but a long term therapy that hopefully will stop and reverse symptoms of Pompe diseast.

This news has long been awaited by the Pompe community. It is also expected that the FDA will soon approve Myozyme in the U.S.

“Read the press release”:http://www.genzyme.com/corp/investors/GENZ%20PR-040306.asp

h3. High School Whiz Kids Search For A Cure For Muscular Dystrophy

While not directly related to Pompe Disease, this is still an interesting piece of news. For a time I was diagnosed as having Muscular Dystrophy before being correctly diagnosed with Pompe disease a few years after.

Cherry Creek High School seniors Lalith Polepeddi and Mike Polmear have found a new approach in finding a cure for Muscular Dystrophy. They are looking at a way to seal leaky muscle cell membranes that plague Muscular Dystrophy patients.

While this research is very preliminary and far from an actual cure scientists are paying attention because of the interesting approach.

Read more about “the story”:http://abcnews.go.com/GMA/Health/story?id=1808757 at the ABC News web site.