December 6, 2012

More on the Cyclodextrin Trial

Below you will find the lastest on the Cyclodextrin Trial at the NIH.  Unfortunately Tylor is not a candidate for this first trial because he is on more than one anti-seizure medication.  We will be following this trial closely and we are hopeful that Tylor will be able to participate in the upcoming trial involving Cyclodextrin.

~ November 28th, 2012 ~

        Cyclodextrin (HP-β-CD) for NPC1 Disease

  ~ Clinical Trial Strategy ~

UPDATE ~ November 28th, 2012 ~ UPDATE
Therapeutics for Rare and Neglected Diseases (TRND)
National Institutes of Health ~ Bethesda, MD 

Dear National Niemann-Pick Disease Foundation Family Members,
The latest release pertaining to the upcoming NIH NPC clinical trial has been made available to the NNPDF. To view the NIH NPC Cyclodextrin Clinical Trial Flyer ~ dated: 11/28/12 ~ click here.
This study titled: 2-hydroxypropyl-B-cyclodextrin (HP-B-CD) in Niemann-Pick Disease, type C1, is in the process of being reviewed by the FDA and the team of researchers and physicians associated with the Therapeutics for Rare and Neglected Disease ~ Niemann-Pick Type C Disease Team (TRND NPC Team) at the NIH are hopeful that they will be able to begin enrolling patients in January of 2013.
The National Niemann-Pick Disease Foundation is pleased that we are able to forward this information along to our family membership. The NIH attached flyer specifies that interested parties should note your interest in possible trial participation by e-mailing a representative at the NIH:
Please note: If you do NOT have access to the internet or an e-mail account please contact the NNPDF Central Offices at the 920-563-0930 and we will assist you in reaching the appropriate contact individual(s) at the NIH for more information.
This is indeed, a very exciting time for all of our NNPDF family community and, more importantly, all of our precious loved ones diagnosed with Niemann-Pick Disease Type C
We WILL Persevere in our Quest for a Cure!
Kind Regards, Nadine M. Hill
Executive Director; National Niemann-Pick Disease Foundation
For a historical timeline on the Cyclodextrin (HP-β-CD) for NPC1 Disease Clinical Strategy ~ outlined by the NNPDF ~ please follow the link above.

What is Blue, Purple and Yellow???

Tylor's eye and cheek!! 

Tylor had a little accident last Thursday.  He had a seizure and fell out of bed, right on the wood floor, face first.  I decided to talk him to the ER to make sure that everything was okay since Tylor can't really communicate if he is in pain or something isn't right. 

They took his vitals and did some blood work to check med levels.  The doctor also order an X-ray to be done.  When he looked at the X-ray and saw no brake but there was fluid present which usually indicates a brake.  So Tylor was then given a CT scan.  Which did indeed show a few fractures to his right cheek bone. Of course there is nothing they can do for this but give some antibiotics to prevent infection and hope that is heals properly.

I am happy to report that the swelling and bruising has gotten much better.  But he doesn't seem himself since the fall. Not sure if it has anything to do with the accident or not but I am watching him closely and hopefully he will start being himself again soon.