The Jett Foundation Fighting Duchenne Muscular Dystrophy

21st Century Cures Passes the House and the Senate

December 7, 2016

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The passing of 21st Century Cures by the United States Senate and House of Representatives is a show of unity and progress for rare disease patients and advocates around the country.
 
Jett Foundation is thrilled about the inclusion of many provisions that will hopefully aid the development of rare disease products. We are especially pleased to see that the Advancing Targeted Therapies for Rare Diseases Act, which clarifies FDA’s authority to leverage data used in a prior targeted drug approval to approve a new therapy that uses a similar or identical technology, was included in this innovative biomedical bill.
 
We very much hope that FDA uses this authority to leverage the data from previously approved targeted therapies, such as eteplirsen, to approve more targeted therapies for Duchenne that also appear to be safe and effective. When you live with a disease that slowly but relentlessly robs you of the ability to climb stairs, walk, take yourself to the bathroom, feed yourself, breathe, and survive, there is no time to waste and every single relevant piece of data should be used by FDA to evaluate a potential treatment and expedite the development and approval process.
 
Thank you Senator Burr, Senator Warren, Senator Hatch, Senator Enzi, and Senator Rubio for co-sponsoring this bill. We hope that someday you witness the result of your efforts living inside a rare disease patient who managed to survive and thrive because of this provision. 

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