Wonderful and Remarkable News for DMD Community

On the surface, it seems self-evident that social collaboration can have a positive impact–especially when it comes to Duchenne muscular dystrophy (DMD).

Working together with all invested participants to influence decision makers in the FDA pipeline world is really about rallying support and engaging with many different folks as part of the process of finding answers.

For those individuals devoted to driving change for anyone affected by DMD, there has been a call to action rally in College Park, Maryland, “We hope to convey to these FDA officials that this is a safe treatment that has a high likelihood to improve the quality of life for the boys who suffer from this tragically progressive disease … we encourage anyone with an interest in helping these boys to talk with these dedicated scientists and parents.”

What is the rally?

The rally was an open communication forum for all invested individuals—to have the opportunity to voice their needs and share their story to influence the release of eteplirsen, “… currently the only drug in the FDA’s approval pipeline for Duchenne, meaning that there are no other short-term prospects for fighting a disease that is one of the most common and devastating genetic diseases of childhood, affecting approximately 1 in 5,000 boys.” Click here to read the complete rally press release.

Why is it important to support the rally?

In today’s FDA pipeline world, learning to work collectively and socially is essential. Gaining the support of multiple decision makers requires connecting with as many invested stakeholders as possible.

teamwork DMD
Teamwork and support really does make the dream work! [Source: pixabay.com]
What were the results of the rally?

In September 2016, the US Food and Drug Administration (FDA) approved eteplirsen (Exondys 51), overruling the recommendations of both its scientific staff and its external advisory committee. Read the published viewpoint press release here.

The best way to gain momentum for a project is to build the core on devoted stakeholders.

Patients with DMD need better treatments, and drugs like eteplirsen might one day fill that role.

Exciting to read about the possibilities of moving in the right direction for the DMD community.


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