You know who DOESN’T need a deadline? Jonah doesn’t need a deadline neither does: Maddie, Pol, Johanna, Elouane, Laura, Levi, Jillian, Lyndsey, Kelsey, Jules, Jared, Charlotte, Kevin, Zack, Bethany, Aimee, Bernardo, Vilho, Veera, Eyra, Hatiam, Khola, Abdullah. Yet all these children were given a prognosis at diagnosis, maybe they might live to their early 20’s but most likely they will succumb in their teens.
Anyone of these Sanfilippo children could die in their sleep tonight for no apparent reason. They could catch pneumonia tomorrow and pass by Christmas. I’m not being mellow dramatic, this is the honest truth.
Can you #Do Something? https://www.crowdrise.com/jonahs-just-begun1/fundraiser/jonahsjustbegunfound
My ask is that you join our team, follow the instructions on the link. I ask that you join the team, because this is much like a pyramid scheme, the larger our reach the more money we raise. However, you can always just donate, I will not hold that against you.
JJB’s online holiday fundraiser #DO Something is hosted by Crowdrise and sponsored by Newmans Own. There are numerous cash prizes given out each week and a grand prize of $500,000.00.
I’m suggesting a December 10th deadline because JJB will be pushing TWO of the weekly crowdrise contests starting on:
-December 12th-December 19th.
The organization to raise the most funds this week will win $10,000
Second place winner will win $5,000.00
-December 31st
The Organization that raises the most funds on the 31st wins $10,000
The Organization that gets the most donations from UNIQUE donors today wins $10,000
We will need you to be ready to push the campaign out to all your contacts on social media by the 12th.
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| Jonah and Laura, France |
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| Khola |
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| Jonah and Juels, France |
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| Pol and Johanna, Barcelona |
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| Jonah and Kelsey |
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| Elouane, Jonah and Laura, France |
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| Pol (Barcelona) and Elouane (France) |
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| Jonah and Ryan Dant |
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| Kevin |
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| & MPSIII C kids all in one backyard, 30 known in U.S. |
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| Jonah and Hatiam |
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| Levi |
A few weeks ago I attended a workshop in California geared towards biotech startup entrepreneurs, It was hosted by Ultragenyx and organized by Springboard. Springboard is a non-profit organization with sites around the world, they advise women entrepreneurs in every type of business sector. Ultragenyx is a biotech company founded by a scientist/physician, Emil Kakkis. As a young postdoc Dr. Kakkis’s research for the treatment of MPSI (JJB focuses on MPSIII) was supported by the family foundation Ryan’s Foundation for Rare Disease Research.
I'm assuming that you already have CNN open in your browser or the news is on in the background. Please put the Trump drama aside for ten minutes and Do Something else. Trump will still be our president tomorrow so you can catch up on the drama later.
I want you to read this story.
http://www.cnn.com/2017/05/12/health/ryan-dant-college-graduation-mps-rare-disease-profile/index.html
My point in having you read this is to remind you that we can create a treatment, we can save Jonah and his friends.
The workshop had experts in the biotech sector that coached the delegates on numerous topics. It was a really great experience and I learned a lot more on the hurdles that we will still need to get through to get a drug passed by the FDA and commercialized. Sadly I was also reminded by the VC’s that MPSIII C will be a major challenge is that we're ultra-rare. I will need to work ten times as hard then even a rare disease to convince an investor to partner with us. I ask myself how am I supposed to do this? I manage two companies and I only have one employee. I’m stretched as thin as I can be. I need more money and lots of it.
The conference delegates were half parents driving the science for their ultra-rare disease and the other half were scientists with a drug candidate that they needed funding for. I didn’t know until the first day of the conference that I was the inspiration for the workshop. One of the BOD of Springboard and employee of Ultragenyx saw me speak at an event. She was moved by my story and wanted to help in someway. She leveraged her connections to make the workshop happen. It’s people like her that make up for what I lack in donations. Pro-bono services from lawyers, grant writers, PR peeps and advisors that have gotten me this far. My lawyer at King and Spalding refused to even tell me how much she charges an hour. I can assume that my bill would have been over $100,000 by now. Sean’s pro-bono grant writing skills have raised Phoenix Nest over two million. What Sean does for our community is priceless. We need more people that have special skills to help parents like myself. I feel guilty that I’m hogging them all. But really should it even be me that has to do all this work? Everyday I want to quit and just hang out with Jonah and my family.
At the workshop it was mentioned by several of the professionals that it takes a certain personality to accomplish what we have. I get annoyed by that, mostly because I know it’s true and I don’t want it to be true. There was a dad in attendance that lost his child at five years old to a horrific syndrome, he has two healthy babies at home. But here he was, out there pitching and working on a treatment. I greatly admired his dedication.
A huge thanks to those that use their special talents and expertise to help people like me and that dad. A huge thanks to those that help by donating.
My patent lawyer is calling in a few. She’s in Denmark with a paying client, yet is taking 30min out of her day to help me. Speaking of which yesterday another pro-bono consultant took the time while on her vacation in Argentina to brainstorm with me on an idea I have, I want to revisit Sanfilippo endpoints for clinical trial. How can we change the status quo of FDA expectations and what measures can we use... Oh yeah then there's Sean who is on his way to Holland, yet yesterday he took the initiative to sign me up for a competition pitch talk. If I’m chosen as the winner they’ll give Phoenix Nest $10,000, Sean wrote my business plan for me and registered me. Then emailed me to mention what he’d done and that I now needed to create a 2 minute video about my company. Did I even agree to this? Whatever, I just cheered myself up thinking about how awesome these people are. Just remembered one more thing… Mari and I had a conference call with a man who specializes in reaching specific markets for his clients. He spent an hour on the phone with us giving us tips and advice that I had no idea even existed on how to market our commercial: Mission Hide and Help. Please watch, participate and share.
This weekend one of my cousins stopped by for dinner, she's from out of town. Andrea asked me: "So what does your Monday look like?" "I know you have these two companies and you're super busy, but what does your day look like?"
To answer your question Andrea.. This is what I did on Tuesday, I can't discuss what I did on Monday NDA required. lol
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| Family, our Aunt Melissa and Grandmother made that quilt. |
Wow I feel so much better. Now if I can only inspire some of you folks to either create a team and promote it on your FB pages, Linkden, Twitter etc. or simply make a donation. Here is the link again. #Do Something
