About Duchenne

Duchenne muscular dystrophy is the most common fatal pediatric disorder. The Duchenne gene is found on the X-chromosome; therefore, it affects mostly males. In rare cases, it also affects females. It affects approximately 1 in every 3,500 live male births. There are approximately 15,000 patients diagnosed with Duchenne alive today in the United States.

Duchenne has no cure.

Duchenne is a progressive neuromuscular disorder that causes a loss of motor, pulmonary, and cardiac function, and ultimately, premature death. It is caused by a mutation in the gene that encodes for dystrophin, a lubricating protein supports muscle fiber strength. When dystrophin is missing in the body, muscle cells are easily damaged, which causes progressive muscle weakness in the entire body.

Children with Duchenne are born seemingly healthy and decline over time, typically losing their ability to walk around the age of 12 and succumbing to the disease in their early to mid-twenties.

Duchenne can be passed from parent to child, but approximately 35% of cases occur because of a random spontaneous mutation. In other words, it can affect anyone.

Two therapies for Duchenne are approved for commercial use in the United States.

One treatment is Emflaza (deflazacort), a corticosteroid similar to prednisone used for decades to reduce inflammation in the muscle and slow the progression of the disease.

The other treatment is Exondys 51, an antisense oligonucleotide indicated for the treatment of Duchenne in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Exondys 51 works by producing a small amount of dystrophin, the protein patients with Duchenne are missing.

For more information on Duchenne, or to learn how to support Jett Foundation’s mission of empowering individuals that are impacted by Duchenne, please contact info@jettfoundation.org.

Diagnosis

We have gathered helpful resources and information for those on a Duchenne journey, in both its highs and lows.

Clinical Trials

For a quick look at enrolling or soon to be enrolling investigational drug studies in Duchenne and their enrollment criteria, check out our Clinical Trial Cheat Sheet.

Treatment & Care

Read more about Exondys 51, Emflaza, Vyondys 53, Viltepso, Amondys 45 and other treatments.

Latest Duchenne News

June 27, 2022
On Saturday, June 25, 2022, Italfarmaco Group shared a press release regarding positive topline data from its completed Phase 3 EPIDYS trial with Givinostat.
June 23, 2022
On June 23, 2022, Sarepta shared news regarding the MOMENTUM study.
Pliant Therapeutics
June 23, 2022
On June 22, 2022, Pliant Therapeutics announced the presentation of pre-clinical data from one of its developing drug candidates, PLN-101325.