Abel Mitja Varela/Getty Images
October 04, 2016 02:37 PM

The U.S. Food and Drug Administration has approved the first drug to treat Duchenne muscular dystrophy, a rare muscle-wasting disease that primarily affects boys.

The genetic disorder causes progressive muscle degeneration and weakness and primarily affects boys, with symptoms appearing between ages 3 and 5, according to the Muscular Dystrophy Association.

Many children with Duchenne begin using wheelchairs between ages 7 and 12. The disorder also affects the heart and lungs, ultimately proving fatal.

The drug that has gained approval, Exondys51 (formerly called Eteplirsen), is designed to combat the disease by increasing production of dystrophin, a muscle-healing protein.

This treatment could potentially triple patients’ lifetimes and increase the number of years they are able to walk.

While Exondys51 treats a genetic mutation that causes 13 percent of Duchenne cases, the approval sets a precedent for the approval of drugs to treat other variations of the disease. Sarepta, the maker of the drug, already has plans to use the same approach to target other mutations of the disease.

“We are excited that, for the first time ever, a drug has been specifically approved for Duchenne,” says M. Carrie Miceli, a scientist who began researching the disease after her son, Dylan, was diagnosed with it in 2004.

“Exondys51 works to repair the genetic defect in some boys living with Duchenne and is among the first examples of personalized genetic medicine in practice,” she tells PEOPLE.

With this approval as a precedent, drugs that use personalized genetic medicine to treat other rare diseases have a clear pathway to approval. More than a dozen drugs to treat other variations of Duchenne are currently in the development and clinical trial phases.


You May Like