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Congressman Dan Donovan

Representing the 11th District of New York

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Donovan, Golden Call on FDA to Expedite Treatment Options for Duchenne Muscular Dystrophy

February 26, 2016
Press Release
Fatal disease currently has no cure; usually results in death before age 25

Brooklyn, NY—February 26, 2016….Congressman Dan Donovan (NY-11) and State Senator Martin J. Golden today called on the Food and Drug Administration (FDA) to expedite its review of treatments for Duchenne muscular dystrophy (DMD). The 100 percent fatal genetic disease progressively breaks down the muscles, usually resulting in death before age 25. No cure exists. Donovan and Golden have worked closely with two local families in advocating for awareness and treatment options for DMD.

Congressman Donovan said, “The children afflicted with Duchenne muscular dystrophy are looking for any hope of leading normal lives. Congress granted the FDA the flexibility to expedite treatment reviews for life-threatening illnesses – the FDA should do so for DMD.”

State Senator Martin J. Golden (R-C-I, Brooklyn) stated, "I have and remain focused on working with families who have young boys with Duchenne Muscular Dystrophy, in an effort to advance awareness and find a cure. DMD is a serious disease that progresses rapidly in young men. I join with Congressman Dan Donovan in urging the United States Federal Drug Administration to make ending DMD a priority on behalf of the more than 40,000 people living with this disease in our Nation."

In a February 17 letter to the FDA, Donovan and other Members of Congress wrote:

 

We urge the agency to fully utilize its authorities and the tools Congress included in the Food and Drug Administration Safety and Innovation Act (FDASIA) to provide for new therapies intended to treat persons with life-threatening and severely-debilitating illnesses…as is the case in Duchenne.

…The accelerated approval pathway outlined [in the legislation] recognizes the limitations of developing drugs for rare diseases and gives the agency the flexibility to grant approval to rare disease treatments that have been shown to be safe and effective in fewer and smaller trials…This allows demonstrably safe therapies that treat an unmet medical need and appear to be efficacious, even with some uncertainty, to avoid the years of regulatory barriers and become accessible earlier to patients who otherwise have no other option.


The full letter is attached.

 

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