What does Exondys 51 Treat?

EXONDYS 51 is the first FDA-approved Duchenne muscular dystrophy treatment for patients who have a confirmed genetic mutation in the dystrophin gene that can be treated by skipping exon 51. In some patients, it helps the body make a shorter form of the dystrophin protein.

Who qualifies for Exondys 51?

Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.

How is Exondys administered?

Administration Instructions EXONDYS 51 is administered via intravenous infusion. Flush the intravenous access line with 0.9% Sodium Chloride Injection, USP, prior to and after infusion. Infuse the diluted EXONDYS 51 solution over 35 to 60 minutes.

What is the cause of Duchenne muscular dystrophy?

Duchenne muscular dystrophy is caused by a genetic problem in producing dystrophin, a protein that protects muscle fibers from breaking down when exposed to enzymes. Duchenne muscular dystrophy mostly affects boys and occurs in one in 3,500 to 5,000 newborns.

Does Exondys 51 work?

Clinical studies have shown that Exondys 51 can improve dystrophin levels in people with DMD. One study looked at muscle dystrophin levels in people before and after receiving Exondys 51 for 48 weeks. There wasn’t a group of people receiving a placebo (a treatment with no active drug) in this study.

Can gene therapy cure muscular dystrophy?

So far, there is no effective treatment but new gene-based therapies are currently being developed with particular noted advances in using conventional gene replacement strategies, RNA-based approaches, or cell-based gene therapy with a main focus on Duchenne muscular dystrophy (DMD).

How effective is Exondys 51?

After 48 weeks, researchers again measured muscle dystrophin levels in people receiving Exondys 51. They found that muscle dystrophin levels were now an average of 0.44% of those in people without DMD.

What are the side effects of eteplirsen?

Eteplirsen side effects

pain, bruising, itching, skin rash, or other irritation where the medicine was injected;
vomiting;
joint pain;
problems with balance; or.
cold symptoms such as stuffy nose, sneezing, sore throat.

Who is most likely to get muscular dystrophy?

Muscular dystrophy occurs in both sexes and in all ages and races. However, the most common variety, Duchenne, usually occurs in young boys. People with a family history of muscular dystrophy are at higher risk of developing the disease or passing it on to their children.

Which parent carries the muscular dystrophy gene?

Duchenne muscular dystrophy is inherited in an X-linked recessive pattern. Males have only one copy of the X chromosome from their mother and one copy of the Y chromosome from their father. If their X chromosome has a DMD gene mutation, they will have Duchenne muscular dystrophy.

How does exon skipping treat a genetic disease?

Exon skipping works like a molecular patch, so that the DMD gene can produce a shorter version of the dystrophin protein to help protect and maintain the strength of muscle fibers.

What is Exondys 51 used to treat?

EXONDYS 51 is used to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 51. EXONDYS 51 was approved under accelerated approval. Accelerated approval allows for drugs to be approved based on a marker…

What is exon 51 (eteplirsen)?

Exondys 51 (eteplirsen), developed by Sarepta Therapeutics, is the first treatment approved by the U.S. Food and Drug Administration (FDA) for a specific group of patients with Duchenne muscular dystrophy (DMD). These patients — 13% of the total Duchenne population — have gene deletions that are amenable to exon 51 skipping.

What is exon 51 used for in Duchenne?