I found this clinical trial on the NINDS (National Institute of Neurological Disorders and Stroke) website – Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease.
This single-patient study includes an 8-month-old male with Type 2 Gaucher disease with progressive neurological decline. From what I can gather, they infused the missing glucocerebrosidase enzyme directly into his brain. I know that Cerezyme doesn’t cross the blood-brain barrier, which is why Gaucher’s Disease Type 2 and Type 3 are ultimately fatal with neurologic degradation.
But I can’t find anywhere to see the results of the study or what happened?! I would love to know what the results were!! If anyone has any suggestions, please let me know.
| Study Start Date: | October 2005 |
| Estimated Study Completion Date: | November 2006 |
This looks like a full article on that trial –
http://jcn.sagepub.com/cgi/reprint/23/10/1231
It looks like more than is on the page you referenced.
I have sent an email to my dad who is a Cardiologist at UCLA. Probably won’t help, but maybe he knows how to get non public info? I will let you know if he can find anything.
I looked briefly and it doesn’t look like they have published any results. I did find this link which give more info about the study itself. http://www.researchcrossroads.org/index.php?option=com_content&view=article&id=128&Itemid=65&trial_id=NCT00244582
I am so glad the other ladies found some additional information. Let us know if it helps, when you get a chance, okay?
BTW: I have an award for you at my blog. You can post it whenever or not! 😀 I just want you to know I am thinking about you.
Petula’s last blog post..On a high note
Carrie,
I know exactly who you are talking about, where he’s from and the details of the whole thing. Aaliyah was a couple days away from being the second child in the world to have the same exact procedure done by the same docs at the same place, things happened and now I’m glad. Call me or email me. Very interesting.
Liz Heinzelman
Aaliyah Heinzelman 7/15/05 ~ 6/26/08
carepages.com (babyaaliyah)
not sure if you still have my # 616.724.4315 or just email me, whichever you prefer. I have been having a very very hard time with the loss of Aaliyah lately. Don’t know if it’s my pregnancy hormones intensifying my already fragile feelings regarding this or if it’s just because I simply miss my little girl.
Liz
HI, “PRINCESS” AALIYAH SENT ME. YOUR PRECIOUS LITTLE HANNAH IS BEAUTIFUL. I WILL BE PRAYING FOR HER AND ASKING JESUS TO HEAL HER. GOD BLESS YOU. LOVE AND HUGS, JOYCE WEISTER