Look at what we started. I have to hand it to our friends at the EveryLife foundation and their work with their program the Rare Disease Legislative Advocates.
Last February, Jonny Lee Miller and I joined a panel of esteemed stake holders in the rare disease community. They spoke to a packed room of […]
The news could not be better! Xavi Hernández, a famous player with, The Barcelona Football Club, and ambassador of, Obra Social La Caixa Foundation, which is a large bank in Spain, has joined the, Sanfilippo Barcelona Association, an association that funds research projects that help in fighting this rare disease.
How are they doing […]
February 28th was, International Rare Disease Day! JJB celebrated the day on Capital Hill with Jonny Lee Miller and friends. JJB’s Jill Wood and Jonny Lee Miller of the CBS’s hit show, ‘Elementary‘, spoke to a standing-room-only group consisting of patient advocates, legislators, and industry, about the importance of supporting federal programs that support […]
We are excited to announce this very special and inspiring Rare Disease Congressional Caucus Briefing
Rare Disease Legislative Advocates, National Organization for Rare Disorders & Jonah’s Just Begun Foundation to Cure Sanfilippo, in coordination with
We are at a pivotal moment for Sanfilippo and the continued evaluation of the soy derived molecule, genistein. While this molecule has been shown to be active in reducing neurodegeneration in preclinical mouse models, so far clinical studies in humans with low concentrations of the natural product have not been successful in showing any clinical […]