Urge Approval of Effective new Treatment for Muscular Dystrophy

children with dmd

Target: U.S. Food and Drug Administration

Goal: Urge the FDA to approve Eteplirsen to treat children with Duchenne Muscular Dystrophy

Today, one out of every 3,600 male births is diagnosed with Duchenne Muscular Dystrophy (DMD). The condition dramatically affects the child’s life, confining him to a wheelchair and subjecting him to a variety of health defects.

Austin and Max Leclaire are two brothers who both suffer from DMD.  While Austin, 14, has been immobile for four years, Max, the younger brother, was selected for a medical trial where he received doses of a drug called Eteplirsen. This drug helps children with DMD to produce proteins and slow down the destruction of their muscles so that they will spend more years of their life mobile.  As a result, Max is still walking around and doing well while his older brother is confined to a wheelchair.

Eteplirsen has so far shown no adverse effects in the children that took the drug, and has improved many young lives by improving their muscular strength and coordination.  However, the drug is not yet available to most children with DMD because it has not yet been approved by the FDA for doctors to prescribe to patients.

The approval of Eteplirsen must be made in a timely manner because the drug can only help DMD children who are still able to walk.  For immobile children like Austin, it is already too late.

Children suffering from DMD across the nation are gradually losing their walking ability right now.  Please sign the petition and help urge the FDA to speed up the drug approval process so that more children can have access to the drug that is key to improving their lives.


To the U.S. Food and Drug Administration:

After following up on news about young Max Leclaire’s participation in the Eteplirsen medical trial to combat his Duchenne Muscular Dystrophy, I was amazed to see how much it had helped him—and how much unfortunate his immobile older brother was from a lack of this treatment.

I would like to urge you to put Eteplirsen through the accelerated approval process and approve the drug in the quickest way possible so that we can improve more lives before it is too late.  The conditions of children with DMD are getting worse each day, and we need to make sure to get Eteplirsen to these kids before they pass the treatable time frame and are confined to a wheelchair for the rest of their lives.


[Your Name Here]

Photo Credits: Justin Ferland Photography via ABCNews

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One Comment

  1. Sheila D GGma Sheila says:

    Our children are our future. We need to do everything within our power to help them emotionally and physically. In a time when mega-corporations such as Monsanto can get barely tested GMOs through the accelerated process, surely we can get Eteplirsen through to help our children. It’s time this administration starts thinking about the people.

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