Duchenne muscular dystrophy (DMD) is a rare disease. Few treatments are available. They may help to slow muscle weakening and loss of motor function, but there is no cure for DMD. Yet, hope is on the horizon:
- National and international health authorities, such as Health Canada, the U.S. Food & Drug Administration (FDA) and European health agencies, are currently evaluating two new therapies.
- A pipeline of new biologic therapies is now in development.
What should you know about treatment?
La Force Foundation encourages parents of children and young adults with DMD to discuss all treatment options that could benefit their children with a competent specialist. Learn more here.
Corticosteroids are the mainstay of treatment for DMD
Corticosteroids help to manage symptoms and improve a child’s quality of life, but they have several side effects. They do not affect the specific genetic mutations that cause DMD.
Experimental treatments are within reach
Health authorities worldwide are currently evaluating the first treatments to target the specific genetic mutations that cause DMD. At present, they include:
- Ataluren targets nonsense mutations that cause DMD in 10% to 13% of children. Learn more here.
- Eterplirsen targets exon 51 skipping, which occurs in 10 to 13% of children with DMD. Learn more here.
What can you do to help us improve access to new treatments?
Take action to help children with DMD to access safe and effective new treatments as soon as possible.