The Jett Foundation Fighting Duchenne Muscular Dystrophy

Jett Foundation Statement: To be Attributed to Christine McSherry, Executive Director, Jett Foundation

April 22, 2016

“On April 25th 2016, in Hyattsville, Maryland a historic event will take place that will change the way people confront barriers to justice in society. This will be historic not just in Duchenne, not just in rare disease, but for every ordinary individual who has felt the need to change a broken system but never knew how to do it.”

“Nearly 1,000 patients and families will convene on Monday. We all represent different continents, countries and states, religions and ideologies. We are all incredibly different, but similar in that we are stewards of hope within the Duchenne Community and among the patients that we serve.”

“The Duchenne community will stand together Monday, understanding that there is impactful and concrete data that supports the accelerated approval of eteplirsen. Together we will ask the data to be viewed, not with a sympathetic eye, but from the scientific bench, as 36 global experts did when they co-authored a recent supportive letter to the agency. In their letter, they agreed that the most ethical and scientifically robust path forward is to grant accelerated approval. The science is there. The safety is there. The approval pathway is also there.”

“For rare diseases, like Duchenne – with an unmet medical need, Congress has worked with FDA to provide tools and authorities that allow the agency to use flexibility when reviewing data for an experimental therapy intended to treat an unmet need, and appears safe and potentially efficacious. Many of these tools, including accelerated approval, were dictated in 2012’s Food and Drug Administration Safety and Innovation Act (FDASIA).”

“Eteplirsen could be the first ever Duchenne therapy in the US, and one of the first mutation specific treatments outside of oncology on the market, leading the way for more Duchenne drugs and precision medicines for other diseases that work by fixing the malfunctioning gene and not simply alleviating the symptoms.”

“Monday presents an opportunity to do more than deliberate the accelerated approval of this drug, and change the course of many other rare diseases in the process. Monday, and our Community’s presence on Monday, will honor the patients who have passed and the families and friends who have mourned their loss. Monday will honor of men and women, boys and girls, who are living with a rare disease today. And Monday could rewrite history for those who do not yet know that they will be affected one day by a rare disease.”

“When I started my foundation in 2001, I thought that the only person who knew how to keep my son alive was me. However, I now realize that our unity is our strength. Rather than existing as separate individuals, our community is coming together as a dynamic unified group that shares similar values, similar fears, and similar dreams for the future of Duchenne, to combine resources and knowledge, and to combat a disease and a barrier to treatment that not a single one person or organization could battle alone.”

“See you Monday, everyone.”



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