The Jett Foundation Fighting Duchenne Muscular Dystrophy

Jett Foundation’s Response to the Duchenne Community on the Accelerated Approval of Eteplirsen

September 19, 2016


Read FDA Approval Letter Here.

Jett jumping Page 1In the face of hardships, in the face of a Duchenne diagnosis, bleak, progressive, and heinous, we as loved one’s of those coping with this catastrophic illness live for littlest of moments. The smallest and tiniest wins in our lives, in our children’s lives, and in the Duchenne Community’s lives, are fragments of normalcy to which we cling while we try to tread water in a tsunami of overwhelming grief and fear.

Today is not one of those tiny wins.  Today is a huge victory for rare disease drug development, for patient input and perseverance, for constructive and educated advocacy, for scientific innovation and integrity, and, most importantly, for Duchenne families.

The Accelerated Approval of the first safe and efficacious therapy for treatment of Duchenne in the United States, eteplirsen, is an event I once never imagined I would witness in Jett’s lifetime. Not only will eteplirsen’s approval change the way that every mom and dad reacts when they hear their child’s diagnosis, it gives hope to an entire generation of children that they too may have the opportunity to live a fuller and more normal life, a life where they can be just like their peers for a little while longer.

All I have left to say is thank you to the people on the front lines and behind the scenes, you who never stopped believing that the approval of eteplirsen was possible… thank you, thank you, thank you…

Jett jumping Page 2-2Thank you to the Duchenne Community, to those amenable and not amenable to eteplirsen, for your perseverance, your hope, and your spirit. Our voices, our children’s voices, were heard through our advocacy, our unwillingness to stand down, and our irrefutable defense of a safe drug that has the potential to save lives. We are still foot soldiers in this war, there is so much work left to do and so many battles left to win. Let’s continue to fight for every child living with Duchenne together.

Thank you to the “Columbus 12” and your families, for your sacrifice, and your bravery in the face of an uncertain experimental drug trial riddled with painful, burdensome, and dangerous procedures all in the harsh light of media scrutiny. Your commitment has ensured that other boys like you have the opportunity to live a longer, fuller lives.

Boys disney worldThank you to the Duchenne scientific and medical community. You are our children’s caregivers and fierce protectors, and your devotion to this community and your patients was never more apparent than on the days you stood before FDA, and proclaimed your unwavering expert support for eteplirsen.

Thank you to my fellow Duchenne organizations and academic institutions who participated in the Make Duchenne History Coalition. We as community leaders became force to be reckoned with when we collaborated with one common goal in mind. Race to Yes, Parent Project Muscular Dystrophy, CDMD at UCLA, and Duchenne Alliance… individually our groups have done remarkable things, but I believe together we achieved what we all once thought was impossible.

Thank you to our allies on Capitol Hill.  Your advocacy for innovation, your constant support for FDA to use all of the scientific and procedural tools at its disposal in the fight to speed safe and effective Duchenne treatments to Rare Diseases Awards Galapatients, was integral to creating this long-awaited day. Your support, your counsel and your friendship is a testament to the goodness that exists in public servants devoted to improving the lives of all of America’s children and families.

Thank you as well to Dr. Janet Woodcock, CDER Director at FDA. Your willingness to listen to patients, to consider and value our input is an example of compassionate leadership that should be encouraged and emulated.

Thank you to Jett Foundation’s supporters. Sixteen years ago when I told the parents on my kid’s soccer team that I was going to save Jett from an incurable disease, you never questioned me, you only asked how you could help. Our supporters love and friendship is immeasurable, and with every donation to Jett Foundation you helped us reach this moment and changed the world’s of thousands of little boy’s just like Jett.

Thank you to Dr. Ed Kaye and Sarepta Therapeutics. Ed, a former colleague of mine from New England Medical Center, was one of the first neurologists I contacted after Jett’s diagnosis and is now courageously leading Sarepta as it breaks barriers and changes Duchenne patients lives. Sarepta has been the Duchenne Community’s loyal partner and friend even when we annoyed you. Your dedication to our community is unprecedented and your team set the
bar sky high for transparency, cooperation, and collaboration. Jett Foundation
looks forward to continuing to work with you in the future.

Another important thank you goes to Chris Garabedian, former CEO of Sarepta Therapeutics. Chris, you are a visionary, one who also believed in a dream that others thought too unreasonable and inconceivable to achieve. Often, in stories like this one, there is a character who falls on swords for the sake of the greater good. Your personal sacrifice, bold and relentless charge towards scientific innovation and regulatory advancement, and unwavering commitment to the patient perspective, significantly contributed to today’s tremendous accomplishment.

Our advocacy work in Duchenne is not even close to finished, in fact it has truly just begun. As long as there is a child living with Duchenne, Jett Foundation will exist to fight their battles big and small. As we the Duchenne Community forge a new path toward a future where tangible hope for a cure for this disease now thrives, the staff here at Jett Foundation will not rest until every child has access to a safe and effective therapy.

For additional comments, please see our media statement

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