A pending FDA decision could determine whether this generation of children is the last to die from Duchenne, or the first generation to live.
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I never thought fatherhood would include things like adding a wheelchair ramp to our home or going on a Make-a-Wish trip. Both of these happened in the last year though. Hospital trips, IEP meetings, and visits with specialists are now also routine.
Both of my sons, ages 9 and 2, have Duchenne Muscular Dystrophy. It is a progressive and fatal neuromuscular disease that affects mostly boys. Life expectancy is short, with most of these boys and young men dying in their teens and early twenties.
There are currently no approved medications to treat Duchenne. There is however a drug in a clinical trial that has been proven safe and effective.
There is a petition on the White House website asking for accelerated approval of this drug. Time waiting for an approval is a luxury we don’t have as my oldest son is already close to losing the ability to walk. We need 100,000 signatures by March 29th.
If you would like more information about our cause, including a link to the petition, please visit theracetoyes.org.
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The Race to Yes webpage explains:
Duchenne is the leading genetic killer of children. The disease affects one out of every 3,500 boys born worldwide. With no approved treatment, Duchenne has a 100 percent fatality rate. A child of five diagnosed with Duchenne experiences a loss of muscle strength leading to wheelchair confinement by adolescence and a shortened life assisted by ventilators. The progressive deterioration of muscle strength leads to death in the late teens or early twenties.
Don’t like ads? Become a supporter and enjoy The Good Men Project ad freeToday, leading scientists see a decisive decade for Duchenne research. The success of landmark clinical trials for the breakthrough drug eteplirsen is a pivotal moment that will lead to the development of additional new treatments for this dread disease. A pending FDA decision could determine whether this generation of children is the last to die from Duchenne, or the first generation to live.
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Sign the petition here.
Photos: The Race to Yes/Facebook