The FDA has just announced an unprecedented decision…
The children with Duchenne muscular dystrophy were heard because it’s “yes” to Eteplirsen!
Eteplirsen was approved! This is absolutely fantastic news, but more importantly, it is a path opening up for other treatments being developed in the fight against Duchenne muscular dystrophy (DMD). Note however that this drug is not a cure and that it only works on the 13% of DMD patients who have the specific mutation.
“The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). 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.” (FDA Press Release)
About eteplirsen
This treatment uses a specific exon-skipping technique to jump over a portion of genetic machinery that produces a non-working, mutated form of dystrophin in children with DMD. Its aim is to restore the machinery’s ability to read genetic code, so it can produce a less mutated form of dystrophin that works in children with DMD.
The production of a partly functional dystrophin may delay muscle destruction and extend mobility in children with this devastating, rare disease. More specifically, eteplirsen triggers the skipping of exon 51, which occurs in 13% of children with DMD.
About Duchenne muscular dystrophy (DMD)
Duchenne muscular dystrophy (DMD) – A degenerative disease of the muscles for which there is no treatment
Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.
Parents and children across Canada: Join the Force…
Our goal is to make Eteplirsen available in Canada as soon as possible!
To subscribe to our newsletter, please step this way.
You can also read these interesting article
