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Monday, November 21, 2016

Woohoooo it's the 22nd of November, no really Woohooo!

How much time do you think you spend: thinking, reading or talking about what Donald Trump just said? It happens to me every morning and I want to kick myself when I realize that I just lost 20 minutes of the first hour of my workday.  Here I am wasting more time again aarg. Please tune everything out and help us out for a second. It will be the best 20 minutes you spent today.

It’s the 22nd, of November our Holiday Campaign: “Save me From MPSIII" has begun.

Haven’t heard of our campaign yet?  Go here. https://www.crowdrise.com/save-me-from-mpsiii

Crowdrise is a fundraising platform that JJB use’s every holiday to raise funds for our research programs. In particular we are fundraising for the last leg of research for our gene therapy program. On March 1st we owe a payment of 77,768.50 GBP.  This past October JJB and our consortium HANDS paid the first payment of 77,768.50GBP.  With that funding our scientist was able to purchase a very expensive piece of software that they will use in this machine (pictured below.)  The software will help guide the needles through little holes drilled into the skull of our children.  We are also using this funding to secure the salary of our most valued postdoc, Claire. Claire will be making the GMP vector which is the drug that will be administered into the brain.  We have practiced on mice and gotten very good distribution results in the brain.  But we want the best distribution that we can possibly get, our children only have one chance at this and it has got to be perfect.  Failure is not an option.  So we will now be testing in sheep, I’m sorry if that makes you queazy and sad, it makes me sad too and I don’t take it lightly.  But remember we’re going to be doing this same operation on Jonah and our friends’s children. If it was your child receiving this experimental drug and invasive operation wouldn’t you want to take every precaution that you could before putting your child through it?  I know it sounds horrific, but for these children the only other option is death.


Last month I went to England to meet with the lead scientist, Brian and his postdoc Claire, they took us through the lab and we got to see everything.  I try to imagine what Brian’s office looks like all the time, what the lab is like, where do our mice live.  Now I know.  We walked over to the hospital and met with the neurosurgeon that will be using the software and preforming the surgeries. Raquel, from Sanfilippo Portugal met me there.  Now that we are so close to trial, we wanted to see for ourselves where our kids would be staying, what they would be going through and who will be taking care of them.  We wanted to meet the doctors and nurses. Put yourself in our shoes, what we’re doing is terrifying, monumental and groundbreaking. Can you imagine our fear and excitement?   We talked to Claire and Brian again about our plans for administration and of course the funding and the next step… trial.



This is it.  This is what we have all been working so hard for.  I hope you can continue to help us get there. I ask those that can use a computer to set up a profile and join our crowdrise team.  Mari will be adding a step by step powerpoint on our website tomorrow. Our campaign will run from November 22nd-January 5th.  There will be several Bonus Challenges throughout the time frame.  We will keep you updated on those dates.  The first one is November 29th, #GivingTuesday, the team to raise the most money on the 29th will win $25,000.  I hope it’s us.

Please share our story with everyone, post your link everywhere, send to all your colleges, clubs, friends and family. You’ll be receiving your JJB 7th annual newsletter in the mail shortly, so don’t feel bad if you don’t use the computer or social media.  We love to receive checks in the mail.  BTW ask your HR department at work if they match donations?! We love two checks in the mail even more.

I hope my fellow Americans enjoy a wonderful Thanksgiving feast.  We sure will, Jeremy is the best cook ever. On Saturday Jonah and I will be joining 6 Sanfilippo families in Paris, for a patient population meeting!  We rented a huge house for all of our families.  I hope they have good insurance.  It’s going to be hysterical!  We will have a french translator to help us explain our research progress, otherwise it would be me, drawing pictures and playing charades. That would be funny too. We always have the best time together.  Ce Bon.
Raquel, the brain guiding machine and Dr. Kamaly

Raquel, Claire, me and Brian

Over the weekend we took Jonah and Sebastion on the Polar Express

Saturday, November 12, 2016

She's got grit

So how about that election…  I kind of had a sinking feeling. I take solace in the fact that Hillary won in the popular vote. I didn’t vote for Hillary because I’m a feminist or because I think Donald is a piece of sh*& but because Hillary is the better person for the job.

Up until now I have never given much thought to being a feminist.  You can call me a feminist and I won’t consider it a label but just one of my attributes. 

I admit I’m a sell out. I haven’t stood up for equality in my mission to create a treatment for Jonah’s syndrome.  When I formed Phoenix Nest, my first thought was, who’s going to be the CEO?  It can’t be me.  Nobody will take me seriously, I’m just a mom, a women.  Asking Sean to stand in front of me as CEO was not a hard decision- he’s a man with a Ph.D even, he’ll be accepted.   My only thought was for Jonah and doing what it took to get a treatment for our kids. Someday maybe I’ll go back and get my MBA and take the helm of Phoenix Nest.

Call me a feminist but don't call me a‘fighter mom’.  People call me a fighter mom all the time, as if it’s a compliment.  For me, as a feminist it’s a derogatory remark.  As if because I’m a women I’ll have to fight the system.  While a man can just make a few phone calls and it’s all taken care of.  You never hear anybody call Mark Dant a fighter dad.

Somebody once told me about a time that they had to try and explain my personality to another person.  This was an awkward conversation for me.   Listening to someone try to sum me up in a word. I stopped her and told her next time you find yourself talking about me, just say: “The girl has got grit.” It has a nice ring to it.

When I found out that Hillary didn’t win office, I was in denial, like watching the trade towers falling- this just can’t be happening.  I couldn’t wrap my brain around it. When it sunk in- my heart  sank.  I thought if Hillary couldn’t convince millions of people that she was the better person for the job.  Then how can I ever expect people to donate to our campaign?  I felt defeated on many levels.

JJB is getting ready to launch our Holiday Giving campaign.  Mari and I have been working very hard on how it is that we’re going to convince people to give this Holiday Season. We talk about what it takes to convince people all the time.  We’re a non-profit we survive off of donations. Yet, it seems impossible to convince people to donate.  Almost a thousand people read my blogs.  Over the last two weeks I have been posting to: Twitter, Instagram and FB asking people to join our: “Save Me From MPS III” campaign.  Mari sent out an email blast. I personally emailed friends and family asking for help. This message hit thousands of people.  In the last two weeks, guess how many people took action and joined our team? 2 people.

I had a little pity party for myself and Hillary earlier this week.  I did things that made me happy- retail therapy helped. I made a collage frame of Jonah and Jeremy’s selfies.  I stare at it constantly, their smiling faces make me so happy. During my down time I realized that I had convinced 100’s of people to do things that they never saw themselves doing.  Case in point, Sean gave up his day job to help me. Our lawyers work pro-bono and discounted rates.  I convinced  the scientists to work on an ultra-rare disease, when they could have been focusing on cancer. I have brilliant advisors around the country that take time out of their busy schedules to help me whenever I ask. I convinced Jonny Lee Miller to help, he ran a 100 miles in one day to help JJB.  I have given talks that have inspired others to follow my lead and who are now creating their own biotech’s and winning grants!  Friends and family from coast to coast (but nobody in the middle…) have given their blood, sweat, and tears to put on fundraisers for JJB.  I have spoken to our congress people and they have been convinced to help us.  It occurred to me that I am good at convincing people to help.  But maybe it’s not completely about convincing people, but reminding them to help.  Reminding them over and over and over again.

So here is your reminder.  Giving Tuesday starts November 22nd, on that day, Crowdrise opens up it’s donation portal.  JJB has set up a campaign on Crowdrise called “Save me from MPS III”
https://www.crowdrise.com/save-me-from-mpsiii/fundraiser/jonahsjustbegunfound
JJB is asking you to donate or join our team. All you have to do is create a little profile and hit the tab on our page that says “fundraise for this campaign” We're asking that you join the team NOW so that we can hit the ground running on November 22nd.  The campaign will run all the way through December. There will be contests and cash prizes that we need to be ready for.

When you link your profile to “Save me From MPS III” you can go in and edit your fundraiser.  Make it personal, upload your own relevant pictures and story of why helping JJB is important to you. For inspiration, read what Jennifer Wood-Mercier wrote.  Then blast the link out to everyone you know and even some that you don’t.

Can you do it?  I’ll be watching.

Wait a second.  It just occurred to me it's INSPIRATION that you all need not convincing!  Duh.  I don't have to convince you that Jonah and the children suffering from MPS are worth saving.  I need to inspire you.  Hm what can I say or do to inspire you?   I need to think on that.  In the meantime look into these engaging, brilliant and happy blue eyes. Jonah doesn't know that he's dying, lets get him a treatment before he does.