What is Duchenne?

What is Duchenne?

Duchenne Muscular Dystrophy, often abbreviated as DMD, is a progressive and severe muscle-wasting condition. Individuals diagnosed with DMD, a form of muscular dystrophy, face significant challenges. By the age of 12, many are reliant on wheelchairs due to muscle degeneration and also encounter social isolation. The condition complicates even the simplest daily activities and, as it progresses, critically affects the heart and respiratory muscles. Duchenne is classified under muscular dystrophies, genetic diseases marked by muscle weakness, with Duchenne being notably the most severe due to the absence of dystrophin, a protein essential for muscle integrity. Research into the genetic underpinnings of Duchenne has been ongoing since the dystrophin gene’s identification in 1986. CureDuchenne is committed to improving the lives of everyone affected through accelerating research to find a cure, improving care and empowering the our community.

Prevalence 

Duchenne impacts around 15,000 individuals in the United States, predominantly males, and over 300,000 globally. It is a condition that transcends cultural, economic, and social lines, affecting families worldwide.

Cardiac Dysfunction 

Adolescence is typically when signs of cardiac dysfunction, a serious disease complication, first become apparent. Nonetheless, maintaining physical activity levels, even among those who are wheelchair-bound, can improve the management of cardiac symptoms.

Advancements in Therapies

Encouragingly, recent years have seen breakthroughs in gene-based therapies targeting DMD. These advancements include gene replacement techniques, RNA-based therapies, and pharmacological interventions, with antisense-mediated exon skipping emerging as a particularly promising approach for addressing the muscular dystrophy caused by Duchenne.

Discover Support and New Treatments for Duchenne

CureDuchenne is at the forefront of providing support and unveiling new therapeutic avenues for those affected by the disease. We invite you to register for our complimentary Caregiver Course, designed to equip caregivers with the knowledge and resources to navigate DMD care. Furthermore, we encourage scheduling a one-on-one consultation with our specialists to explore how these and other treatments can make a positive impact.

Explore Clinical Trials for DMD

Stay informed about the latest clinical trials and explore our impactful work in the field of Duchenne Muscular Dystrophy.

Newly Diagnosed?

CureDuchenne is a resource you can rely on. We have parents on staff who have children living with Duchenne, a team with years of experience working with families affected by Duchenne, the top medical and scientific advisors, and a vast network of families working together to support each other. Please reach out to us if you need assistance or have any questions. We can help. We want to help. It’s the reason we exist. Learn more and contact us HERE.