Treatment approaches and disease management

Multiple treatments working together can address the many effects of Duchenne and help preserve function.

No single treatment addresses every aspect of Duchenne

There are several approved therapies available to help manage Duchenne. In some cases, healthcare providers may use more than one therapy to help support muscle function over time. Supportive treatments may also help manage heart and lung health, mobility, and other symptoms.

Your healthcare team can help determine which treatment approach may be right for you.

Since Duchenne causes multiple changes in muscle cells that contribute to muscle damage, therapies are designed to target these changes in various ways:

Flame icon with a diagonal line through it, illustrating steroids

Steroids^reference

Steroids aim to reduce inflammation in the muscles.

They are often used as a starting point in Duchenne care. Steroids can be used regardless of genetic variant.

Steroids^reference
Steroids aim to reduce inflammation in the muscles.
They are often used as a starting point in Duchenne care. Steroids can be used regardless of genetic variant.
Gene therapy^reference
Gene therapy is designed to help the body make a shorter version of dystrophin.
Not everyone is able to receive gene therapy, and doctors will run certain tests, like checking for antibodies in the blood, to see if it’s an option. Gene therapy does not fully replace dystrophin.
Exon-skipping therapies^reference
Exon-skipping therapies help the body make a shorter form of dystrophin by working around certain genetic changes.
This treatment can only be used in patients with specific genetic variants. Exon skippers do not fully replace dystrophin.
HDAC inhibitor^reference
An HDAC inhibitor is designed to reduce HDAC overactivity, which occurs when dystrophin is missing.
An HDAC inhibitor can be used regardless of genetic variant.

Talking to your doctor

Talking with your care team can help you better understand available treatment options and how different approaches may fit into an individualized care plan.

The following questions to ask your doctor can be downloaded and taken with you to your next appointment. Use the button below to create your document.

Here are some questions about treatment options for Duchenne that you can ask to get the conversation started:

Should I consider using more than one type of treatment?
Could my genetic variant impact which types of treatments are right for me?
Beyond treatments that target dystrophin, what other approaches should I consider?
What other important factors should I consider when deciding which treatments are right for me?
What should I know about the benefits and risks of combining multiple treatments?
How might insurance coverage, approvals, or out-of-pocket costs affect my treatment options?

Have more questions? Enter any other questions you’d like to discuss with your doctor below.

I understand that any questions and concerns around side effects should be discussed with my healthcare provider.

I acknowledge that these questions will be downloaded to my device as a PDF. The information I submit will not be saved or shared.

Treatment approaches and disease management

No single treatment addresses every aspect of Duchenne

Steroidsreference

Talking to your doctor

Get more information about Duchenne and learn about a treatment option.

Steroids^reference