INDIANAPOLIS (WISH) -- It's a devastating diagnosis that changes a child's life forever, Duchenne muscular dystrophy, or DMD, is a rare and fatal genetic disease that primarily affects boys.

It slowly robs them of their ability to walk, breathe, and live independently.

There have been no treatments targeting the root cause, only ways to manage the symptoms. But now, a breakthrough could change their lives forever.

Pediatric neurologist Omer Abdul Hamid at Nemours Children's Hospital says there are new therapies that target the root cause of DMD.

"Now we have new options that actually address the underlying cause of the disease, the missing protein. We can give a gene therapy or what we call gene transfer therapy, where we actually transplant the missing gene into the body," he said.

It isn't a cure, but it may slow the damage.

"It should slow the progression, and hopefully, actually help these boys regenerate some muscle," he said.

Gene therapy for DMD offers hope, but it comes with the need for close monitoring, especially in the first three months. Patients may require frequent lab work, hospital visits, and steroid adjustments.

Though some have experienced serious side effects -- those cases have been successfully managed, with patients making full recoveries.