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First-of-its-kind gene therapy could change everything for Hoosier children

Duchenne Muscular Dystrophy gene therapy was approved by the FDA in June
Posted at 10:36 AM, Oct 26, 2023
and last updated 2023-10-26 10:36:29-04

INDIANAPOLIS — The first gene infusion therapy of it's kind in the state was performed at Riley Hospital for Children this week.

The Duchenne Muscular Dystrophy (DMD) gene therapy was approved by the FDA in June.

DMD is a genetic disorder that causes muscle cells to fall apart.

Without treatment the life expectancy of a patient with DMD is late teens or early 20s.

With the current steroid treatment it can be into their early 30s.

Almost every patient stops walking around 10 years old.

"It's one of those diseases that we've always had to just manage and help families through and we always knew the course," Dr. Laurence Walsh from Riley said.

However, the new kind of therapy, approved by the FDA could change all of that.

"This drug treatment gives us a chance to alter that course and hopefully for a good way for a long time," Walsh said.

The gene therapy will help stabilize the muscles, Riley is the first hospital in the state to do it.

WRTV was able to speak with Paul Jr., the first child to receive the treatment, and his family.


Paul Jr.'s family says it a saving grace for them

"Every time you have a kid you think of all the things they're going to do in their future and all the stuff they like and get to pursue," Paul's mom said.

The long term impact of the treatment is still unclear but doctors are hopeful it will give patients a longer life and one that isn't so debilitating.