Author Archives: zeighami

The Unlocking Lifesaving Treatment Act of 2012 (HR 3737) is giving the rare disease world new hope.  Across the country, people with rare diseases (roughly 10% of the population) and others are coming together at the grassroots level to show … Continue reading →

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 →

Did you know: More people, collectively, suffer from a rare disease than HIV + AIDs combined Accelerated Approval has only been used once for a rare disease treatment (since 1992) Accelerated Approval has been used dozens of times for HIV … Continue reading →

I believe that the rare disease community is on the verge of a revolutionary shift.  What once was a weakness has become a strength.  Our community is spread far and wide by its very nature.  This broad distribution of people … Continue reading →

Most days I just go about my daily business.  Of course I am always aware of my son’s illness and it rarely leaves my mind but most days I am not bitter.  I don’t often shake my fist at God … Continue reading →

I wanted to take just a short minute so crystallize a thought.  As I read stories on the FDA and drug approval, I am struck by the way consumers/patient groups are characterized.  There is an impression that is given that … Continue reading →