Monthly Archives: December 2011

Patient advocates have amazing power.  The 1983 Orphan Drug Act enabled an entire industry treating rare diseases.  However, the fact is it wasn’t enough.  A huge portion of the 7000+ rare diseases are still without treatment. This upcoming year, a … Continue reading →

If you can keep your head when all about you Are losing theirs and blaming it on you; If you can trust yourself when all men doubt you, But make allowance for their doubting too: If you can wait and … Continue reading →

Just a short note highlighting our trip to Geneva for Sanfilippo Congress.  Sorry for any typos, I am blasting this out fast. I have tried my best to relay the information as I received it.  I am not a scientist … Continue reading →

Dear EveryLife Foundation for Rare Diseases, Thanks for your support of ultra-orphan diseases. I am 100% behind this effort because its result would be tangible and measurable – namely how many rare diseases are granted accelerated approval based on a … Continue reading →