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, breathe 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

25July
25 July @ 01:00 pm - 02:00 pm

Update on Sarepta’s RNA-targeted technology platforms (PMO and PPMO)

Webinar details Dr. Gilmore O’Neill, Chief Medical Officer of Sarepta Therapeutics, Inc, will give a scientific overview of Sarepta’s RNA-targeted exon skipping technologies, PMO and PPMO. Dr. O’Neill will highlight the PPMO technology, designed to potentially improve upon the levels of dystrophin produced by PMO-induced exon skipping, and to potentially lead to more efficient dosing […]

24August
24 August @ 08:30 am - 04:30 pm

Continuing Education | Duchenne: Current Understanding and Treatment – Baltimore, MD

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

25August
25 August @ 08:00 am - 03:00 pm

Family & Caregiver Workshop – Baltimore, MD

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.

07September
07 September @ 05:30 pm - 09:00 pm

8th Annual Getzlaf Golf Shootout

The 8th 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.

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8th Annual Getzlaf Golf Shootout

The 8th 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
50 Monarch Beach Resort North
21June
Learn More

DUCHENNE ARTICLES

ANAHEIM, Calif., Nov. 15, 2017 /PRNewswire/ — Boys and young men in advanced stages of Duchenne muscular dystrophy experienced significant and sustained improvements in cardiac structure and function, as well as skeletal muscle function, following treatment with CAP-1002, the lead investigational therapy under development at Capricor Therapeutics (NASDAQ: CAPR). These findings were reported today by […]

CURE DUCHENNE NEWS

PTC Therapeutics Update on Ataluren Study 041 PTC Therapeutics is enrolling boys and young men with a nonsense mutation in a clinical trial for Ataluren.  To ensure that participants who enroll in Study 041 get the most benefit, and to ensure their safety, there are strict criteria around who is able to enter the study. […]

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