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Dec. 23, 2014

TOPIC: Medical, Patients & Members, Advocacy, Industry, Patient Stories

Saving Eliza

Posted by admin

I’m honored to be speaking at the NORD Breakthrough Summit on Rare Diseases and Orphan Products in October.   Social media, as I continue to learn more every day, is an always changing, rapidly moving, fickle and funny world.  Trends can get hot and can come and go overnight, and others go on and on (ALS).  Our journey for the last year has been to advocate and fundraise for Sanfilippo Syndrome, with which my 4-year-old daughter Eliza was diagnosed.  This rapidly degenerative, terminal disease has no treatment to slow it, and no cure..but a clinical trial is potentially only months away.  We’ve been fortunate to have some ideas take off and be successful in the social media world, like the 3- minute viral video at, which raised over $1M in less than 3 months.  At the NORD Summit, I will talk about how that came to be and lessons learned along the way.

I will also talk about our latest project.  The ALS Ice Bucket Challenge success was a wake-up call for all fundraising, of just how connected we all can be, through the simple power of sharing.  After hearing the first report of ALS raising $1M over just one weekend, we launched our #Sing2Lines for #SavingEliza challenge campaign to stop Sanfilippo, at We wanted to take a shot, and set ourselves apart as different.  It’s catching on, and we hope it can bring awareness and much needed funding for Sanfilippo.  We will talk about how we launched that and, by the time of the talk, we can give an update on progress.

I’m also going to talk about some advice received along the way, and how we’ve adapted to social media.  This will include things like 1) Start with the story of one  2) Never mention the word Sanfilippo..wait, what?  3) You (before diagnosis) are your audience.

There are great people in this world who want to help all of our causes.  They just don’t know about us.  We have to make it clear and easy for them in the crowded social media world.  Creativity, validation, and a sense of timing seem to be some key elements that help set the successful campaigns apart. I look forward to sharing our experiences in the hope it can help other foundations in their efforts.