1st

Drug approved by the FDA
for the treatment of Duchenne.
CureDuchenne was an early funder.

9

CureDuchenne funded research projects
have advanced to human clinical trials

BE A MEMBER OF OUR PATIENT REGISTRY

Enroll here to make sure you’re alerted and aware of the latest pharmaceutical treatments for Duchenne muscular dystrophy that become available as research progresses every day.

Our lives were changed forever…

“We didn’t know anything about Duchenne and felt isolated that there was no one we could turn to… CureDuchenne has hosted several workshops that have been invaluable in providing knowledge on the care, treatment and future of our son, but, even more importantly, has enabled us to spend time with other Duchenne families…”

READ FAMILY STORY

Duchenne is a devastating muscle disease.

It’s the most common and severe form of muscular dystrophy. Those affected with Duchenne lose their ability to walk, feed themselves, breath independently and succumb to heart failure. But there’s hope through new pharmacological and gene-based therapies. You can help make a difference.

LEARN ABOUT THE DISEASE

UPCOMING EVENTS, WORKSHOPS & WEBINARS

04March
04 March @ 04:30 pm - 10:00 pm

Napa in Newport

Napa in Newport benefits CureDuchenne, a local organization with global impact, aimed at saving the lives of children with Duchenne muscular dystrophy.

08April
08 April @ 08:30 am - 02:00 pm

San Diego, CA – Workshop for Duchenne Families

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy.

08April
08 April @ 07:00 pm - 11:00 pm

2017 Champions to CureDuchenne – Austin

Join fellow community and business leaders in the fight to cure Duchenne during a fun and memorable evening of great food and drinks, entertainment, live and silent auction.

21April
21 April @ 08:30 am - 04:30 pm

Milwaukee, WI – Duchenne: Current Understanding and Treatment

Details for this event are being finalized. If you would like to reserve your spot today, please email Celeste@cureduchenne.org or call 949-872-2552.

1 2

Napa in Newport

Napa in Newport benefits CureDuchenne, a local organization with global impact, aimed at saving the lives of children with Duchenne muscular dystrophy.

04:30 pm - 10:00 pm
1 Ritz Carlton Drive
04March
Learn More

DUCHENNE ARTICLES

FDA Approves EMFLAZA™ (deflazacort) Tablets and Oral Suspension for the Treatment of Duchenne Muscular Dystrophy in Patients 5 Years and Older

Articles
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Marathon Pharmaceuticals, LLC (Marathon), a U.S. research-based biopharmaceutical company focused solely on the development of new treatments for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted approval of EMFLAZA™ (deflazacort) for the treatment of Duchenne muscular dystrophy in patients 5 years and older. Duchenne, a severe form of muscular dystrophy, is a rare disease and fatal genetic disorder that affects about 15,000 people in the United States.1

CURE DUCHENNE NEWS

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

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