Here is an example in Duchenne MD of why I spent last week at the FDA. In a degenerative disease, if a drug can maintain abilities, why do children have to continue to die while the company proves what we already know in a larger group of kids AND with a placebo group of kids who WON’T get the drug??? What Sarepta tried to avoid in seeking Accelerated Approval for their Duchenne drug is what we are facing in Hunter Syndrome.
It kills my heart that kids with Hunter Syndrome who do not qualify to enter the clinical trial will continue to die for the next probably 3-4 years while we await the Phase III trial and drug approval.
I do my level best to impact the FDA for our boys.