KINGSTON, Mass., April 22, 2016 /PRNewswire-USNewswire/ -- "On April 25th, 2016, in Hyattsville, Maryland a historic event will take place that will change the way people confront injustices in their 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 communities and the patients that we serve."
"The Duchenne community will stand together Monday, understanding that there is impactful and concrete data that supports 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 just more than grant approval to a drug, and change the course of many other diseases. It will honor the patients who have passed and for their 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, know I 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."
About Duchenne muscular dystrophy: Duchenne is a progressive neuromuscular disorder that causes a loss of motor, pulmonary, and cardiac function, and premature death. It is the most common lethal pediatric genetic disorder, and it affects every 1 in 3,500 live male births and some females. Duchenne is caused by the body's inability to create dystrophin, a large protein found in muscle cells. Duchenne has no FDA approved treatment or cure and is 100% fatal. Children with Duchenne are born normal and decline over time, usually losing their ability to walk around the age of 12 and succumbing to the disease in their early to mid-twenties.
About the Jett Foundation: Since 2001, Jett Foundation, located in Kingston, MA, has worked to find treatments and a cure for Duchenne muscular dystrophy (DMD) while improving the lives of those affected by DMD.
The Jett Foundation is a registered charity with 501(c)(3) status from the IRS; all donations are tax deductible. www.jettfoundation.org.
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SOURCE Jett Foundation