My BRAVE Story: Urgent message to the FDA

Dear PPMD and FDA,

Like many parents have, I'll start by giving my son's age. Miles is 6 years old and in 1st grade. The significance of age is obvious to all of us. It is a reliable marker of where he is in a journey with a tragic arc, where every day, month and year counts.

Miles is a happy kid. At 6, he is able to walk and play, participate in most activities at school and at home, dress himself, travel, ride the bus and swim in the ocean. That's not to say he doesn't have challenges - he has a cognitive delay, has never been able to run or jump, and is now old enough to start to feel the difference between his abilities and those of other kids. Still, life is sweet, for now. I believe that is partly because as parents we have striven to convey a sense of optimism and excitement in life. As every child should, he holds the expectation of a bright future. I wish we could freeze time and stay in our current reality forever.

That brings me to what I dread most. Miles doesn't yet know that in many ways, life is (may be?) better now than it will ever be for him again. I dread the day that brutal reality sinks in to his consciousness-just the recognition of inevitable, brutal, crushing decline and loss of abilities feels unbearable. I would do almost anything to prevent or postpone that moment of terrible recognition.I fear that as much as the actual physical challenges.

But, I know that without help, it's coming-sometime in the next few years, I imagine. Each day that passes will see him lose a little more of his capability, mobility, stamina until eventually there will be no denying the trajectory. My heart goes out to all the parents who have sat with their child through that horrible moment or series of small realizations that amount to the same. I know that wouldn't be the end - life and hope and joy would still go on. And, I know this is an old story that keeps repeating itself in family after family.

Now, for some of our families, there is the opportunity to break that cycle and banish the weight of inevitability that hangs over us. Thanks to the incredible breakthroughs in medical research, we can change the story for many of our boys! There is hope for a much brighter future... miraculously there but maddeningly just out of our reach.

Miles is incredibly lucky to have an exon deletion that makes him a candidate for drisapersin and eteplirsen. He was in a 6 month drisapersin trial and in the low dose arm. Everyone, not just his parents but researchers, family and friends, saw him get stronger with the drug. For the first time in his life, he was running and (almost) jumping. We believe it also affected his learning - while we can't be sure of the cause, during the trial his language skills made a big jump. It was a wonderful time of hope and progress.

Then, the door slammed shut. It's been over 6 months since the trial ended. He hasn't jumped or run since, and is more tired than he was then. We understand the questions about efficacy of drisapersin but we sincerely believe that this happened.

Miles would also be a candidate for eteplirsen. We are ready to go wherever and whenever we need to get it for him. We would drop our jobs, sell our house, move to another country. Because we know what will happen if he doesn't get the medication, we know it's terrible, we know every passing day is a closed door. And we're just like all the parents with kids in this position.

Every day that we delay costs our children. Sure, my son can hang on for a year, he'll probably still be walking in 2 or 3. Longer? Well, we understand what abilities he will lose - it's the natural progression of DMD.

We deeply appreciate that there are dedicated professionals looking out for all of us. But, this is a special case where the risks of moving the trial ahead more quickly are far less than the risks of inaction.


I'd like to hear from the FDA what parents and advocacy groups can do to provide the cover and support they need to make these life saving drugs available now, not years from now.

Thank you,
Elliott Barnett

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