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

08September
08 September @ 08:30 am - 04:30 pm

Duchenne: Current Understanding and Treatment – St. Louis, MO

This course is ideal for physical therapists, occupational therapists, speech language pathologists, and others involved in the care of persons with Duchenne.

08September
08 September @ 05:30 pm - 09:00 pm

7th Annual Getzlaf Golf Shootout

The 7th Annual Getzlaf Golf Shootout – hosted by Ryan Getzlaf, captain of the Anaheim Ducks – is a two-day charity golf event that brings together athletes, celebrities and community leaders in support of CureDuchenne.

09September
09 September @ 08:30 am - 02:00 pm

St. Louis – Workshop for Duchenne Families

Please join CureDuchenne Cares for a free, informative class on Duchenne muscular dystrophy, focused on educating parents and caregivers on the best standard of care for Duchenne management.

22September
22 September @ 08:30 am - 04:30 pm

Duchenne: Current Understanding and Treatment – Indianapolis, IN

This course is ideal for physical therapists, occupational therapists, speech language pathologists, and others involved in the care of persons with Duchenne.

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7th Annual Getzlaf Golf Shootout

The 7th Annual Getzlaf Golf Shootout – hosted by Ryan Getzlaf, captain of the Anaheim Ducks – is a two-day charity golf event that brings together athletes, celebrities and community leaders in support of CureDuchenne.

05:30 pm - 09:00 pm
08September
Learn More

DUCHENNE ARTICLES

Data show for the first time a systemic therapeutic effect in DMD dogs using a rAAV2/8 micro-dystrophin gene therapy approach without immunosuppressive treatment CAMBRIDGE, Mass., July 27, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today […]

CURE DUCHENNE NEWS

NEWPORT BEACH, Calif., June 15, 2017 – RASRx announced today that the United States Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) for its compound RASRx1902 for the treatment of Duchenne Muscular Dystrophy.  RASRx1902 is an oral therapy that has shown positive effects on muscle function in animal models of Duchenne.  […]

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