Jenn McNary on Fox’s Stossel Show – we will get results!

Posted by: Christine McSherry on March 21, 2014


Please remember to circle Thursday, March 27 on your calendar.  That’s the night that Fox News will air John Stossel’s interview with Jenn McNary.  (It’s just before of our deadline of March 29 for 100,000 signatures on our White House petition…but more on that in a minute!)

The Fox producers will have creative license to decide what goes into the final program, of course, but it will be impossible for them to miss Jenn’s key message.  I was with her in New York City today for the taping, and she never wavered from her core message: the FDA has all the data it needs to grant accelerated approval for eteplirsen, and begin saving the lives of thousands of children (primarily boys) children (approximately 1% of those diagnosed are girls, many moms are carriers who exhibit some symptoms – especially in their hearts, some female siblings are manifesting carriers.)

Joining Jenn in the Fox studio were Darcy Olsen of the Goldwater Institute and former Ohio Congressman Dennis Kucinich.  Olsen was squarely in our corner and frowned on the FDA continuing to dragggggg its feet on approving a drug that has ZERO safety issues and DOCUMENTED clinical benefits for our DMD boys.  Regardless of where you stand on politics, you have to applaud the Goldwater Institute for standing up for the proper way to run a government by the people and FOR the people.

I was mildly surprised about Kucinich — and it had nothing to do with his politics.  He looked Stossel in the eye and stated that the FDA has an obligation to (paraphrasing here) “protect the public.”  If you hear someone snorting off to the side on the television set, that was me.

PROTECT THE PUBLIC? SERIOUSLY??  Let me offer a three-part rebuttal.
First, we’re talking about Duchenne Muscular Dystrophy here, the No. 1 genetic killer of young boys, and some girls. Before eteplirsen, there has never been a drug that has shown ANY clinical benefit for DMD boys.  If Kucinich believes the FDA is correct in trying to protect my son from eteplirsen, he is very much misguided.  Without this drug, my son Jett will die — as will thousands of others like him. Eteplirsen has a stellar safety profile.  In the clinical trial that has gone on for two and half years now, there HAS NOT BEEN ONE ADVERSE SIDE EFFECT.  The drug is safe. Truly, it’s that simple.

Second rebuttal: eteplirsen has produced dystrophin in 100% of the patients in its study, including Jenn’s son Max.  Her other son — Austin — did not participate in the study, and he is now confined to a wheelchair and continues to lose motor control and decline.  Please, Mr. Kucinich, put yourself in Austin’s position — and watch your brother Max continue to thrive on eteplirsen while your own body wastes away. Austin is 15 years old now, and he’s very wise to the workings of the world.  He is well aware that there is a drug available that could save his life, and he cannot comprehend why the United States government denies him access to it. Perhaps you can explain that to him, Congressman?

My third rebuttal speaks for EVERY boy with Duchenne.  If there is a drug that shows even a “hint” of efficacy, WE WANT IT NOW. This is a progressive neuromuscular disease, and without an effective treatment, boys will continue to wither and die.  Some parents of those with Duchenne have questioned why Jenn and I do not “play nice” with the FDA and let things run through the proper channels.  My blunt answer never changes: WE HAVE NO CHOICE.  If we don’t start dosing eteplirsen to those who suffer from Duchenne immediately, we will lose the chance to stop the progression of the disease.  And once that window closes, it’s gone — forever — and we need to make the FDA understand that there is ZERO justification for delaying access to eteplirsen.

OK, back to the White House petition.  As I am writing this, we have secured almost 60,000 of the 100,000 signatures needed before we can put our issue before President Obama. This awesome level of support puts us within reach of our deadline to complete the petition by March 29.  (If you’re reading this, you’ve probably signed up. But have you asked your co-workers and your college classmates to sign up?  Please, please, please: take a minute and try to add at least one more name to our list!)

Our petition drive has a  clear objective: to shine a very bright light on the realities of eteplirsen and the psychology of the entire Duchenne community.

Even as an experienced Registered Nurse, and Jenn without a medical background – but a world of experience thanks to having two children with the disease, and we struggle to explain the science behind the morpholino chemistry that makes eteplirsen so effective. But we are parents of children who have Duchenne, and we are first-hand witnesses to what this drug can do. This drug has halted these boys from declining – and even more miraculously – it has stabilized, and in some cases improved their pulmonary function. 120 week data was recently presented by the company, as a parent, and a nurse – I was astonished (and very elated) to see that MIP and MEP (measures used in pulmonary function) were stable. THIS NEVER HAPPENS IN DUCHENNE – we know that respiratory function begins to decrease even before ambulation is lost – to have this stabilization means that we truly have something life altering for the non-ambulatory population! This should have the FDA spinning in their chairs to use the tools they have; FDASIA, Accelerated Approval – to get this drug out to those who will benefit today!

In closing, join me in three cheers to John Stossel for covering our story, and don’t forget to watch his program on March 27. PLEASE encourage your friends, family and associates to sign our petition at TheRaceToYes.Org.  And finally, please close your eyes for a second and imagine yourself as the parent of a boy with Duchenne. We are facing a killer disease, and we have a drug that is safe and effective.  Ironically, the FDA sees itself as protecting us but in fact it is decidedly NOT protecting our children from the ravages of Duchenne. We need an FDA that can listen to its congressional mandate, and open the doors to the miracles of eteplirsen. We have nothing to lose — literally — and everything to gain.

We are so close we can taste it.  We can save this generation of  boys with Duchenne.  And we don’t have a single second to waste.

On behalf of Jenn and the ENTIRE family of those with Duchenne, thank you for joining our fight. Like the Goldwater Institute says, “results matter” — and with your help, we can get the results we need to help our boys beat this dreadful disease.

Link to Video of Jenn McNary on John Stossel Show: HERE

link to Video of Jenn and Christine on The Willis Report - Fox Business:  HERE

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