LAST NIGHT. WE DID IT! THANK YOU TO ALL WHO SIGNED AND SHARED.
WHAT A MONUMENTAL NUMBER!
Keep an eye out for more media coverage, more news, more progress. We have a long way to go to get the dosing started but at least we now have hope that the voices of all these brave parents and their children will be heard.
This is a very similar situation to the Dallas Buyers Club story, only Ryan's medicine isn't available in any other country. It IS available here yet the FDA won't grant accelerated approval. The FDA has data that shows its safe. The FDA has had this data from clinical trials for over 18 months and done nothing. Its time.
THE RACE TO YES CONTINUES...