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Genzyme's Horizon Newsletter
I had my first official interview regarding Hannah and Gaucher’s 2/3 Friday afternoon.
A reporter from Genzyme’s (the people who make Cerezyme, Hannah’s enzyme replacement therapy) called to get “Hannah’s story” for their upcoming newsletter, Horizons. This newsletter will go out to the entire Gaucher community (types 1, 2, and 3), clinicians, etc.
It was a very easy process, as the reporter was very genuinely interested and easy to talk to. He explained the first part of the article will be an explanation of GD23, and the second part would be about Hannah and our fight for her.
What was most interesting (but not surprising to say the least), the reporter admitted that this is the first GD23 story that he has done. I have a feeling that it is possibly the first GD23 story to be in one of their newsletters!
I don’t think Genzyme was trying to shy away from GD23, but GD1 is really all that has been touched on in their previous newsletters. Like with so many other Gaucher type 1 families (95% are type 1), our form of the disease is kind of like the “forgotten” type of Gaucher’s since there are so few of us. But Genzyme has really been awesome in trying to help me connect with other families and in trying to get our story out there.
The reporter is going to send me a copy of it before it goes to print. I really hope it gets people’s attention to realize that our children (Hannah and the other GD23 kids) are fighting a losing battle right now, and that we really need their help to save our children’s lives!

Dr. William Ondo, Associate Director of the Parkinson Disease Center at Baylor, Houston
About a month ago, I sent an email to the Baylor Department of Neurology’s Parkinson’s group asking if I could meet with one of their clinicians to have Hannah’s symptoms evaluated. I explained that Hannah has neuronopathic Gaucher’s disease, and I wanted to talk with someone regarding the possible commonalities between the two diseases.
Yesterday afternoon, I got a phone call from the scheduler for this department saying that Dr. William Ondo, Associate Director of the Parkinson Disease Center and Movement Disorders Clinic is willing to consult with us and evaluate Hannah! Because she is a patient at Texas Children’s Hospital (Baylor’s pediatric hospital), all of her records and patient information was already accessible to him.
We meet with him in early July, right before we leave for NIH. The timing for this appointment is actually perfect, even though it is about 5 or 6 weeks away because it gives me more time to learn more about the disease processes of Gauchers and Parkinson’s.
After meeting with the occupational therapist this week and watching Hannah playing with her toys, I realize that we need to get her some more “advanced” toys for her birthday in July. All of the ones she has are very, very basic, and that she is ready for some more interactive toys that will help her develop her fine and gross motor skills.
So I went shopping on Toys R Us online tonight, and I went through at least 600 to 700 toys. Out of all of those, I only found 11 toys that I thought would be really beneficial and helpful to Hannah. That is it! Of course, I am no therapist, so I’m hoping that some of my special needs moms can share their opinions on these toys. I put them all together on a list, so I can do some more research into these. If you have any familiarity with these, please do share your thoughts, good and bad!











When we went to see Hannah’s ENT doctor a week or so ago, we were in the elevator on the way down, and one of the doctors going down said “Hey, your daughter doesn’t blink.” He didn’t say it in a concerned way, just more like he found it somewhat amusing that she kept her eyes wide open the entire way down.
So I had experimented with her for the rest of the afternoon. Sure enough, she can go for over 10+ minutes without blinking. I got tired of watching at that point! She isn’t having a seizure or anything, as she just goes about her day when this happens, playing with toys, drinking a bottle, taking a walk, etc. She has no problems interacting and has no changes in her personality when this happens. We also have no idea when this started because we had never noticed it before!
She will close her eyes if you touch her eyelash, and when she gets tired she will close her eyes (most of the way) to sleep.
So we shared this at our pediatrician visit, and he was just amazed at this symptom. So we now give her eye drops a couple of times a day to make sure her eyes are lubricated.
This is where it gets very interesting…
I asked Dr. Sidransky at the NIH about this new symptom, and her response was “That is very interesting and not typical of the eye movements in type 3 GD. It will be interesting to see what our neuro ophthalmologist thinks!”
I did some research into this, and you know what disease lack of blinking is a symptom of? PARKINSON’S DISEASE! Check it out, 1st on this list from MedicinePlus Encyclopedia/NIH. You can NOT tell me that these two diseases do not have a definite commonality of some sort in their disease process!
Hannah, who has a never-before-seen genetic mutation of neuronopathic Gaucher’s disease, has developed a symptom that is not a symptom of her Gaucher’s disease but IS a symptom of Parkinson’s disease, the very same disease that last week a 10-year-study and a second study solidifed the genetic link between the two diseases.
There is a VERY STRONG connection here!! Something that DESERVES to be looked at! Hannah and the other GD23 children truly MAY hold a key to understanding Parkinson’s disease, yet we can’t seem to get them to notice!
http://rarediseases.info.nih.gov/TRND/
The need and opportunity for Therapeutics for Rare and Neglected Diseases (TRND) are enormous. Of the 7,000 human diseases, fewer than 300 are of interest to the biopharmaceutical industry, due to limited prevalence and/or commercial potential. More than 6,000 of these diseases are rare (defined by the Orphan Drug Act as <200,000 U.S. prevalence), and the remainder are neglected because they affect impoverished or disenfranchised populations. Researchers have now defined the genetic basis of more than 2,000 rare diseases and identified potential drug targets for many rare and neglected diseases (RND).
TRND received $24 million in the National Institutes of Health (NIH) budget for fiscal year 2009. TRND is a collaborative drug discovery and development program with governance and oversight provided by the Office of Rare Diseases Research (ORDR). Program operations will be within the intramural research program adjacent to the NIH Chemical Genomics Center (NCGC) and will be administered by the National Human Genome Research Institute (NHGRI).
Frequently Asked Questions
TRND (PDF – 30)
Rare Diseases (PDF – 21KB)
Neglected Diseases (PDF – 36KB)
News
TRND Press Release (PDF – 80KB)
http://news.smh.com.au/breaking-news-national/cell-malfunction-linked-to-many-diseases-20090521-bfv7.html
The tiny “recycling unit” at the core of every human cell can fail, and research is increasingly placing this malfunction at the root of a host of common deadly illnesses.
Alzheimer’s disease, stroke, heart disease and certain cancers can all be linked to a dysfunction of the lysosome, says South Australian biochemical geneticist Professor John Hopwood.
“You might think these lysosomal diseases are uncommon but in fact we’ve been studying the tip of an iceberg,” Prof Hopwood says.
“The more we study these disorders … it turns out the lysosome has a big role to play in many illnesses that the community has.”
Lysosomal disease is a process which sees affected human cells lose their ability to create new versions of themselves, instead becoming clogged as genetic “material gets into the recycler but can’t get out”, Prof Hopwood explains.
His team at Adelaide’s Women’s and Children’s Hospital led the development of world-first treatments for two rare lysosomal diseases – Maroteaux-Lamy and Hunter syndromes.
These and other Lysosomal disease occur in one in every thousand children and it results in developmental delays, bone deformities, heart and breathing difficulties, behavioural problems and a shortened life span.
Prof Hopwood has also developed a test able to highlight these genetic conditions in newborns, allowing treatment to get underway before irreversible features develop.
While his work has focused on treating children with these rare conditions, Prof Hopwood says the field’s future would also go to unearthing the links between lysosomal disorders and common diseases.
In the brain, the disorder was known to lead to the loss of brain matter which, over time, could manifest as Alzheimer’s or dementia.
“Where you have heart failure because a valve doesn’t function properly, it may be due to poor signalling by control systems that are influenced by these lysosomal storage disease problems,” Prof Hopwood says.
“So these rare diseases give us an insight into the ‘berg’ part of the ‘iceberg’, which is affecting the majority of us.
“And if we can understand how it contributes then we can reduce the impact of all of these disorders in the community.”
The Australian Academy of Technological Sciences and Engineering (ATSE) paid tribute to Prof Hopwood for his 25 years work in the field at a gala event in Sydney on Wednesday night.
He was announced as one of the recipients of a 2009 ATSE Clunies Ross Award, which recognises the nation’s pre-eminent scientists who have bridged the gap between research and the marketplace.
Just like my friend, Heather, and her husband, Jim, helping us raise money for Hannah’s fight (Makdan publishing), my friend, Debra, has offered to donate 30% of all sales of her absolutely adorable teddy bears and stuffed animals for Hannah’s cause!
For my Canadian friends, please take a moment and consider Bears N Buddies. She has been around for awhile, and she has such a huge heart. She donates often to causes she cares about, and I want to thank her and support her anyway I can!
See the cute teddy bears and stuffed animal kits! Thank you, Debra, for everything!
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