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Thursday, June 14, 2012

Double edged sword.

After Jeremy and Jonah sang me Happy Birthday, Jonah proclaimed "my turn!"Just happy to oblige.
So annoying! My yahoo account was hacked. I changed the PW and that seemed to stop it.  Sorry for the spam.  It’s bad enough that I naturally spam my friends, only to have someone else hack in and do it for me, well that’s just overkill.

My life is a double edged sword, catch 22, damn if you do damn if you don’t.  The future is terrifying.  I’m glad to be a year older and closer to the finish line.  Time means that Jonah is getting older too and closer to brain damage.  His 4th Birthday is just 6 weeks away.  Two years ago, when we started this campaign we assumed he’d have brain damage by now.  He doesn’t!  Now we "assume" that we will have another 2 years before he starts to change (based on observance of other MPSIIIC kids.)

For the most part our business plan and executive summary for Phoenix Nest is done and I’m ready to hit Boston with it.  I feel like it’s finals week, Ive done all the cramming I can do I’m just waiting for the test.  Monday can’t get here fast enough!

While researching for PN, I found a few articles discussing the most expensive drugs in the World.  I was relieved to find that out of the 12 most expensive drugs in the world, five of them are for Lysosomal Storage Diseases (LSD’s) which Sanfilippo is.   Sanfilippo is also part of the MPS’s (Mucopolysaccharidosis.)  2 of the 5 LSD treatments are for MPSI and MPSII.  I was relieved because... I’m scared.

I’m scared that I won’t be able to convince a venture capitalist (VC) to invest in Phoenix Nest.  I created our virtual-biotech out of necessity, our patient population isn’t large enough for a big drug company to take interest in us. Our alternative is to do it ourselves, for that I need a financial partner.  But why would a VC want to help an ultra rare disease company?  Well because, we can charge the World’s highest prices for a Sanfilippo drug.    Phew- I can breathe a little easier now!

Then it occurred to me...  I have just become one of those evil capitalists, a company that my neighbors occupied Wall St. to shame. Ugh! But what am I supposed to do?  Sit back and watch our children die?!  Because I don’t want to over charge for a treatment that I have to have to save my son?!  A treatment that would never see the light of day if it wasn’t for the parent advocate groups driving the science....

I’m sorry I don’t have a billion dollars to do it all by myself!!!!  I have to convince someone that does into helping us. I can’t apologize for being happy that we can charge exorbanent amounts of money.  Life is a double edged sword. On a side note most of the manufactures of these expensive drugs give scholarships for those that don’t have insurance.

So here is my compromise. On the slim chance that we do get VC backing and Phoenix Nest is able to discover and create a treatment that in turn makes millions and millions of dollars.  I’m going to open up a private nonprofit school for special needs kids (Phoenix Nest Refuge.)  The school will be the best, a parents dream come true and free of charge.  We’ll shame the other greedy capitalists into helping us maintain the school!

After entering the realm of the special needs world, I have met many amazing parents with wonderful kids that need help.  Our government is turning their backs on our kids.  If there isn’t a special program in our district, then our government forces us to mainstream our kids.  This is totally unexceptable, for everyone involved. So here we put a child who may not be able to communicate, walk,  sit still for more then two min.’s and is prone to outbursts- in a classroom with normal kids.  Who’s benefiting from this?  NOBODY! Not the parent, child or his classmates not to mention the administrators and the rest of the fall out effect. What a disaster.

Many parents are forced to move to different districts in hopes of getting their children into a special needs program, only to pay thousands of dollars for tuition. It’s added insult to injury, our lives are complicated enough!  We're already bankrupt from having to pay through the nose for medical expenses.   As it stands now, our governments is saying to us: “Your child will never contribute to society, so make him a ward of the state; we’ll warehouse him for you until they die and are no longer be a burden on you and society.”     (fu)

So I fantasize about creating a beautiful school with all the amenities that our special needs kids need.  Because our children deserve nothing but the best, they deserve the same quality of life as anybody else, IF NOT MORE!  Who's making all the sacrifices here?

The odds are stacked against Jonah and this generation of rare disease children with no treatments.  There are 7,000 rare diseases and only 5% have treatments, 75% of the rare disease affect children. :( BTW the definition of  a rare disease is one that afflicts 1 in 200,000 live births.  MPSIIIC affects 1 in 1.4 million.

To make all my dreams come true I need money and lots of it.   Money will never by anyone happiness but it will buy our kids a cure...  Fingers crossed I can convince a VC to give some funds to Phoenix Nest.  In the mean time JJB needs to keep our payment plan for our gene therapy.  On September 1st JJB has to pull together $90K for our third payment for the first ever treatment for MPSIIIC  :) I'm very proud of this.

I have until June 16th to raise a 2k from the text to Rare program.  JJB's 1k will be matched by the EveryLife foundation.  On top of that my friends Dad has pledged to math another $340.00 (the remainder, needed to make the 1K)  I created a birthday wish, sent out an email blast, tweeted and blogged about this fundraiser.  I can't believe we haven't gotten a 100 supporters to text rare to the number 85944.  This just says to me either nobody reads my cries for help or people just don't get it?!  If you can afford 10$ and text please do it now. We only have until the 16th! Go here now. http://www.everylifefoundation.org/texttodonate.aspx