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Donate Now!
Please help Jonah on his journey towards a cure! Click the "Donate"
button above to contribute via PayPal. Or, send a check to: Jonah's Just Begun - Foundation To Cure Sanfilippo, Inc. PO Box 150057 Brooklyn, NY 11215-9997 Jonah’s Just Begun – Foundation To Cure Sanfilippo, Inc. is a New York Not-For-Profit
that was formed to help raise awareness of this disease. The Not-For-Profit receives and then distributes funds for scientific,
educational and charitable purposes as described in section 501(c)(3) of the Internal Revenue Code. Jonah’s Just
Begun – Foundation To Cure Sanfilippo, Inc. has applied for formal recognition of tax exempt status under section 501(c)(3)
of the Internal Revenue Code. We expect the formal recognition of tax exempt status to be effective as of the date of
formation of the Not-For-Profit, so we will gladly accept monetary donations by check or Paypal. Our formal determination
letter for the Not-For-Profit status was granted December 2010. Also, please see our merchandise advertising Jonah's website on Cafe Press. The items here do not provide us with funds, they are simply meant to be worn and used as tools to raise awareness.
Help us raise awareness by sporting a fashionable and "green" tote bag to the grocery store. Send your tot off
to preschool in Jonah's logo tee. Wear your t-shirt to your child's next sporting event and be sure to bring your
reusable water bottle! When Jonah grows up he will need a healthy environment to live in. Research for Sanfilippo subtype C is currently in the works! Jonah's Just
Begun - Foundation to Cure Sanfilippo Inc. is thrilled to have started funding the work of Dr. Alexey V. Pshezhetsky. Alexey was able to turn the modest grant of $20,000 that JJB gave him in the fall of 2010 into seed money. Alexey
in turn won a grant from the Canadian Institute of Health for $650,000. This grant has given Alexey four years of funding
and facilitated the start our first ever type C mouse model. JJB's next funding venture will be with doctors Daniel
Grinberg and Lluisa Vilageliu. JJB’s third project — a joint venture with doctors Alexey Pshezhetsky and Brian
Bigger — will follow. By 2013 we hope to be funding one of the current TFEB projects as well. Our foundation is also in the process of recruiting other Sanfilippo subtype C children to participate in our Natural History Study. We have helped families abroad with medical costs incurred during their participation in the retrospective Natural History Study. JJB is currently reviewing proposals for subtype C’s NHS. Our four research projects plus our NHS will run from 2-4 years and cost around two million dollars. This may sound like a lot of money to some. But once you break it down over the course of four years, then factor in the possibility of outside funding from the National Institute of Health or a drug company. It then becomes very feasible (once our researchers have proved some efficacy and shown the validity of the project, it is very likely that JJB will receive outside help.) It’s also important to remember that JJB is not the only foundation raising funds for subtype C research. Our sister foundations: Sanfilippo Sud (France) and Sanfilippo Barcelona (Spain) are doing their part as well. Board directors Raquel Marques & Antonio Vincente are also working hard raising funds and awareness in Portugal. The Sanfilippo community is fortunate to have several notable researchers searching for treatments for our children. Just a few years ago this list was less then half. Today we are very fortunate to have so many researchers and clinical doctors working on cures for our children: Alexey Pshezhetsky, Brian Bigger, Simon Jones, Haiyan Fu, Maria Escolar, Matt Ellinwood, John Hopwood, Daniel Grinberg, Luisa Vilageliu, Olivier Danos, Jean Michel Heard, Adrea Ballabio, Marco Sardiello, Alessandro Fraldi, Zacharon, Frits Wijburg, Gregorz Wegrzyn, Elisabeth Neufeld, Jerome Ausille, Chet Whitley and Elsa Shapiro. These researchers are in need of continued funding. Once parent advocate groups like JJB do their part in driving research, the drug companies will step in. The incentives of the "Orphan Drug" act give drug companies more of a reason to develop drugs for rare diseases. With the current changes in big pharma's finances — losing their large patents like Lipitor — drug companies have found that they will need to expand their research departments, Pfizer, for instance, just added its own orphan disease department. Thank you so much for your support! Your generosity is what is going to save Jonah — and many more children— from slowly slipping away from us and succumbing to this insidious disease. Thank you! You can always support Jonah and his journey via the Jonah's Just Begun Cause page on Facebook! If you have a birthday coming up, consider creating a "Birthday Wish" in honor of Jonah's Just Begun. It's easy; follow the link to JJB's Cause page and click on "Wish."
Raquel (left) and Jill (center) with researchers Matt
Ellinwood, Alexey Pshezhetsky and Brian Bigger, at the Sanfilippo Workshop in Northampton, UK. Matt Ellinwood does toxicology testing on our Sanfilippo animals. |
Jonah's Just Begun - Foundation to Cure Sanfilippo, Inc.