Drug approved by the FDA
for the treatment of Duchenne.
CureDuchenne was an early funder.
CureDuchenne funded research projects
have advanced to human clinical trials
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…”
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.
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. […]