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

NEWPORT BEACH, Calif.–(BUSINESS WIRE)–#DuchenneCureDuchenne
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
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Contacts

CureDuchenne
Karen Harley, 949-872-2552