CureDuchenne is excited to share that NS Pharma is launching expanded access for viltolarsen, allowing patients with DMD amenable to exon 53 skipping to receive this product while it is being reviewed by the FDA. CureDuchenne thanks NS Pharma for showing dedication to the DMD community by opening access to this treatment merely weeks after FDA’s […]
CureDuchenne Helps Duchenne Families Make Informed Decisions About Durable Medical Equipment with New Interactive Website
NEWPORT BEACH, Calif. (September 10, 2019) – Finding the right mobility equipment for Duchenne muscular dystrophy patients can be a time intensive and frustrating process. To help take the guesswork and hours of research out of the equation for families and caregivers, CureDuchenne announced today it has launched a comprehensive interactive website called the “Durable […]
The nonprofit CureDuchenne has chosen veteran advocacy leader Erin Frey to become its first director of Duchenne muscular dystrophy (DMD) advancement efforts. Read the full article on Muscular Dystrophy News.
More than $900,000 will go to support CureDuchenne’s mission to find a cure for Duchenne muscular dystrophy and support patients and families affected by this rare genetic disease, all thanks to Orange County’s wine connoisseurs, philanthropists and business leaders who gathered March 2 for the fifth annual Napa in Newport to benefit the nonprofit. Read the […]
Scientists are testing nearly two dozen treatments that might stop the disease. But enrollment in the trials is very restricted, and few children qualify. Lucas was 5 before his parents, Bill and Marci Barton of Grand Haven, Mich., finally got an explanation for his difficulties for standing up or climbing stairs. The diagnosis: muscular dystrophy. […]
CureDuchenne, one of the nation’s leading Duchenne muscular dystrophy (DMD) charities, is sponsoring 30 events across the U.S. this year to educate patients and their families about all aspects of the neuromuscular disease. “Out motto is ‘leave no boy behind,’ so we want to make sure we have a format that works in smaller, less […]
In last year’s action film “Rampage,” which featured Dwayne “The Rock” Johnson, CRISPR gene-editing technology turns a gorilla, a wolf, and a crocodile into monsters the size of skyscrapers that destroy Chicago. If you missed the movie, you may have caught wind of He Jiankui. Near the end of 2018, the Chinese scientist reported having used CRISPR […]
When it comes to Duchenne muscular dystrophy (DMD), Leonardo Feder knows he’s among the lucky ones. A resident of São Paulo, Brazil’s largest city, Feder was diagnosed with Duchenne at age 6 and immediately began taking corticosteroids as well as heart medicine and supplements. “I’ve been well-medicated all my life, so I’m fine,” the 33-year-old said […]
In November 2016, two San Diego families received the same devastating news about their little boys. Four-year-old Yannick Etienne and 5-year-old Marko Filenko were each diagnosed with Duchenne muscular dystrophy, a progressive muscle-wasting disease that would one day take their lives. Read the full article here.