My BRAVE Story: A drug that could help him feels so close yet so far away

My beautiful son Connor: Is 9 years old Needs exon 53 skipped Can pass the six minute walk test Just got his first power chair (loves it). A drug that could help him feels so close yet so far away. I can taste it. I can pray. I can hope. Yet I find myself thinking if only he had been born now maybe there might be a drug available to help him when he needs it. Then, I think of everyone who was born ten years before my son. There were not the possibilities then that we have now. How do they feel?

My son is at such a critical point. Just maybe he will get into the Sarepta trial this year (if that still is going to happen) where they will compare him to those on eteplirsen then roll him into a trial to skip exon 53. That will take a year at least. By then he might not pass the six minute walk test. If he can, he might be on placebo. At the rate things are going, we are looking at 5 or more years until a drug is out. He will be 14 which means his big chance is up. Time, time, time. There isn't any if you want our sons to have a chance. Then we have the issue of deflazacort. A drug that is considered better than prednisone for muscular dystrophy (especially since the side effects are less) all over the world except in the US. The reason I hear it is not used here is that the company that makes it does not want to spend the small fortune it would take to have it approved in the US. Since I have the resources to get it, my son can benefit from its use. What about all the others who can not afford it? The world is a global economy. It is time to think of medicines in those terms. There is no reason for our regulatory agencies not to be able to collaborate with other governments. The focus should be saving lives and better quality of life not the laws and self interest of our country.

I understand some of the difficulties the FDA has on an intellectual/patient safety level, but on a personal/family level my husband and I are willing to take a bigger risk because time is the killer. We understand all that the FDA does for patients. In addition, we especially appreciate that you listen to our community and are trying to rethink your expectations for trials with rare diseases.

Thank you for all you do!

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