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Wednesday, January 9, 2013

Revved up and ready to go.

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Holy Snow!
Revved up and Ready to go. 

Nothing like the fear of greedy insurance companies to get my blood boiling.

Here are two recent articles that have freaked me out, I’m not going to panic…. Yet. If Jonah was already receiving an expensive treatment I’d be taking up arms now.  MPS patients that are receiving monthly infusions of enzyme should be getting ready for battle now.



Insurance companies are beginning to scrutinize the health benefits that patients get from using expensive drugs.  Basically they don’t want to pay for quality of life.  Why is it ok that health care coverage is a billion dollar industry for insurance executives?  Jonah’s Physical Therapist nailed it: “People aren’t M&M’s”- Jane Weedon of WorkSpace.

These articles have given me invaluable information and have validated my concerns.  This generation of patient pioneers have to protect our children and the generations of Sanfilippo children to come, we will have to justify to insurance companies as to why our children need expensive drugs and treatments. A well designed and executed Natural History Study is a must.  The Natural History Study will paint the picture of doom and gloom… the nightmare that our children and their families are living in.  Much like a page ripped from a Stephen King novel.  

The MPSIIIC gene therapy research that JJB is funding, will cost over a million dollars per child (just one time.) Insurance companies won’t like that and many families will have to fight for it.

The article's have also given me another reason as to why we can’t put all of our eggs in one basket and focus on just one treatment.  We need to think outside the box and find treatments that aren’t going to cost a fortune.   It’s a fine line for an ultra rare disease.  A treatment has to be expensive enough for a drug company to find as a worth while investment, but if it’s too expensive insurance companies will put up a fight.

JJB will hope for the best but plan for the worst. 

Don't read into this blog.  I'm extremely optimistic for the New Year.
I’m super excited about another project that JJB and JLK are going to fund. It’s an edgy new concept that has never been done before.  The experiment has been in the works for over a year now and preliminary results are optimistic, so we’re going to jump in.  Not only is it a noninvasive and potentially inexpensive treatment but it’s application for other Lysosomal Diseases are promising.   

JJB’s funding for this project has come straight from the generous donations from the Holiday Remit fund.

I can’t even begin to thank all of our friends and supporters that have donated (I especially like our friends that work for companies that have matching programs, you’ll all be in my will.)  Donations from $5.00 to $500.00 it all adds up.   I watched this ridiculously stupid movie over the Holidays. The main character was bitching about the dent put into her $10,000 dollar couch.  I wanted to puke… What I could do with $10,000!  Seed money that has the potential to save thousands of lives, not to mention the unmeasurable difference in the quality of life that it would make for the affected child’s entire family.

Rare disease affects 1 in 10 people.  You know why my family is probably the only family you know of?  The majority of the other families are shut in’s.  As soon as the parents get home from work their child’s caregiver is relieved and they take over the constant care.  They don’t leave the house for dates or vacation, they can’t afford too.  Who will care for their child and how will they pay for it? There is no disposable income after paying for expensive medical care.

So for those of you who are embarrassed that you only have $5.00 bucks to donate to the Holiday Remit Fund, don’t be. We’re a testament to what five bucks can do. 

Next week I'll tell you more about how your donations have changed scientific history.  Alas, you'll have to wait.  I have to pick Jonah up from school now. 

Happy New Year!
Jill