September 7 is World Duchenne Awareness Day, a global day of recognition for the one in 3,500 newborn boys who live with Duchenne muscular dystrophy (DMD).
MDA knows that urgently needed treatments and cures for this devastating disease can’t come soon enough. That’s why we have invested more than $200 million in DMD research. MDA has been central to the development of exon skipping from the beginning in the 1990s, funding foundational work upon which the approach was built as well as extensive research into the strategy since that time. MDA supported the early development of eteplirsen — the very first FDA-approved treatment for DMD — via funding to Steve Wilton, then at the University of Western Australia in Perth, who pioneered the exon skipping technique that allows eteplirsen to work.
Read more about MDA’s work in finding a cure for DMD and meet some of our incredible families who are living testaments to the progress we are fighting for everyday.
Five Questions with DMD Researcher Tejvir Khurana
Five Questions with DMD Researcher James Novak
Jerry Lewis’ Work Helped Scientists Discover the DMD Gene
DMD Treatment Update: Emflaza [Webinar Recap]
Access to Health Care and to Approved Therapies is Essential for MDA Families
Living with DMD:
Light It Up Green for Muscular Dystrophy Today!
For the Love of Devin: Josh Argall Races to Find a Cure for His Son — and All MDA Families
Montana boy first in the state to receive Exondys 51
Caregivers Give Back: Parents of Son with DMD Offer Advice for Others, Raise Money for MDA
The Rumble of Motorcycles Means Hope for One Pennsylvania Family
Devoted Father Raises More Than $50,000 for Team Momentum
Sharing is Caring: Why the Krieger Family Walks
Help us continue to fund Duchenne research.