CureDuchenne Applauds the FDA Approval of Marathon’s EMFLAZA (deflazacort) for the Treatment of Duchenne Muscular Dystrophy

EMFLAZA is the First Drug in the U.S. Approved for Use by Patients Age Five and Older with Duchenne Regardless of Genetic Mutation

 CureDuchenne applauds the U.S. Food and Drug Administration (FDA)  approval of EMFLAZA™ (deflazacort), developed by Marathon Pharmaceuticals, the first drug in the U.S. approved for use by patients five and older with Duchenne muscular dystrophy regardless of genetic mutation.

EMFLAZA is a corticosteroid that demonstrates anti-inflammatory and immunosuppressant effects. Clinically, EMFLAZA has meaningful benefits in treating patients with Duchenne. Those treated with EMFLAZA showed improved muscle strength and showed slower decline in functional ability. Learn more about EMFLAZA here.

“For the first time all those impacted by Duchenne muscular dystrophy have a FDA-approved treatment available to them,” said Debra Miller, Founder and CEO of CureDuchenne.  “This is a milestone for the Duchenne community to have access to this therapy that shows improved muscle strength and slower decline in functional ability.  As a mother of a young man with Duchenne, deflazcort has been an important part of my son’s standard of care since he was diagnosed. We are delighted that Marathon Pharmaceuticals is committed to providing access to the drug with a comprehensive patient assistance program.” 

Duchenne is a fatal genetic disease that causes muscle degeneration.  Patients are usually diagnosed by age 5, most lose their ability to walk in the teens and most don’t survive their mid-20s. 

“This treatment provides hope for families impacted by Duchenne,” said Miller.  “We need to continue to extend and improve the lives of those affected by Duchenne by funding research that will help treat all aspects of the disease until there is a cure.”     

About CureDuchenne
CureDuchenne is the leading nonprofit focused on funding research to find a cure for Duchenne muscular dystrophy, a disease that affects more than 300,000 boys worldwide.  CureDuchenne’s mission is to extend and improve the lives of those affected by Duchenne. With support from CureDuchenne, nine research projects have advanced to human clinical trials.  CureDuchenne provides the Duchenne community with resources on the best standard of care through its CureDuchenne Cares program. For more information, please visit CureDuchenne.org and follow us on Facebook, Twitter, Instagram and YouTube.