Team Sanfilippo

I have some very exciting news to share with all of you.  A collaborative between Sanfilippo families and researchers has been formed to find a viable treatment option and cure for our kids.  Here is an excerpt from the website, to give you some information:


Rare diseases, including lysosomal diseases, typically receive less attention in both basic research and therapy development than common disorders.   Team Sanfilippo is a research collaborative initiated by a group of family-based foundations to accelerate therapy development for Sanfilippo Syndrome (Mucopolysaccharidoses III).  This effort is conceived as a research collaborative consisting of scientists at academic, industrial and government institutions conducting basic, translational and clinical research on MPSIII disease.  The specific objective for which the families anticipate providing funding is to develop, within a three-year period, a potential therapy that can be tested clinically to prevent or significantly delay the onset and progress of clinical neurological disease in MPSIII patients.  The envisaged Collaborative will conduct regular teleconferences to share and discuss research findings and set objectives.  The parent-funders will conduct regular calls as well, and the entire group will meet in person at least annually.  Each of the scientists in the Collaborative will bring unique expertise which fosters collaboration between the laboratories and enhances research efforts towards the common goal of developing treatments for MPSIII disease. The research will be focused on three areas necessary for identifying potential therapies suitable for clinical trials:  High throughput screening for drug identification, animal models for drug testing, and biomarker identification.
Accomplishments to date include the identification and retention of a Project Manager with extensive pharmaceutical industry experience who has been successfully working on a similar collaborative project involving another lysosomal storage disease (Niemann Pick Type C).  As a first step in forwarding the objectives of Team Sanfilippo, a scientific meeting has been scheduled for November, 2009 to review the status of research and development related to MPSIII disease.  Attending this meeting will be a group of more than a dozen leading scientists from academic, government (including the FDA and NIH), and industrial institutions willing to share their knowledge and willingness to help formulate a strategic plan for achieving the Team Sanfilippo objectives.


Matt and I are very excited about this project.  Our geneticist from Children's Hospital, who is also a director with NIH, is going to attend the conference.  I am eager to hear her thoughts and ideas when she returns.  If you have any interest in supporting this effort, please leave a comment or email me and I can give you details.  We are all hoping to raise money to pool together to support this cause.  There has been a non-profit set up to receive donations specifically for Team Sanfilippo.


More information can be found at the Team Sanfilippo website:  http://teamsanfilippo.org/

Comments

Jaime Arnett said…
Hi Shannon, my name is Jaime Arnett. I have been very close friends with a friend of yours Christina Scott for about 10 years and she recently told me your families story. I have been brought to tears MANY times reading your families story, it sounds silly, but i feel like i know you all. I think about your sweet family every day and i my two older children are getting to know your sweet babies through pictures. They cant wait to see how Waverly and Oliver are doing and we all are praying for you every day. Sorry this is such a long comment...I just wanted you to know how much you and your family have touched our lives. I would love to know more about this new research and any way that i cant give my support, if you could include my e-mail on a list jaimearnett@gmail.com Thank you for sharing your life and touching so many others lives in the procces! ~jaime
Misty said…
Shannon, Thanks so much for getting the wor dout. We need all the support we can get 100% of all proceeds will go directly to research. We have been fundingthe project manager and are funding this meeting and will need more inthe future buit that is part of thr research initiative. The "donate now" key should be up soon. I also am sedning all of our funds to Team Sanfilippo, so you could diect support to our paypal account on our site. 100% of our funds are directed tothis project as well. misty
Jenkins said…
That is very cool!
Hopefully there will be some great progress made to helping those with Sanflippo.
Jenkins said…
Sanfilippo. I spelled it wrong...spelling is not my favorite thing...

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