The Final Countdown
Gene Therapy have you ever heard of it? Sanfilippo Syndrome have you ever heard of it? If No, than consider yourselves fortunate.The children and families suffering from rare genetic diseases are not so fortunate. Parents of terminally ill children love their children everyday as if each day could be their last. Sanfilippo Syndrome is a horrible genetic […]
JJB Joining forces with Cure Sanfilippo Foundation
Release Date: 11/26/2018 Jonah’s Just Begun to join forces with Cure Sanfilippo Foundation Jonah’s Just Begun (JJB) and Cure Sanfilippo Foundation are pleased to announce that they will be joining forces as they move forward with the common goal of finding a cure or treatment for Sanfilippo Syndrome. Formed as a 501c3 in 2011 by […]
Canceled- Gingerbread House of Horrors
In light of everything that has gone down the past few weeks, we are calling off our Halloween fundraiser Gingerbread House of Horror. We hope to postpone to next year. Mari is working on that now. Jonah is not a happy camper about this wheelchair thing, it has unleashed the lion in him, and that is […]
Insight Into Sanfilippo Provided by ConnectMPS Global Registry
We spent a few days in San Diego at the International MPS Conference hosted by the MPS Society. Alongside our ConnectMPS and Mission: Hide and Help posters was a poster published by BioMarin. This is a perfect example of how sharing patient-reported data can speed up the drug development process when shared freely. Click on image to […]
“Compared to what Sanfilippo Children and their Families have to go through, a Tough Mudder seems like Nothing” -Mari-Elaina Garcia (JJB’s Director of Operations) Mari has lost her mind and decided to participate in the Tough Mudder Half on July 22nd, just days before her 39th Birthday. In lieu of gifts, she is asking for […]
