Ohio sets the trend in screening newborns for rare disease. What to know about DMD

Mariyam Muhammad, Columbus Dispatch

Ohio Gov. Mike DeWine announced that the state will be first in the nation to screen all newborns for a disease called Duchenne Muscular Dystrophy (DMD).

“Adding DMD to the list of screenings newborns receive ensures that parents will be able to start initiating care early for children who are diagnosed with this condition,” DeWine said in a release on the state government's website.

DMD was added to the list of 40 other rare medical conditions in the Ohio Department of Health’s Newborn Screening program in HB 33, the state's budget for the fiscal years 2024-25. DeWine signed the bill in 2023. From estimations, the Ohio Department of Health expects that screening will identify 35 babies with DMD in Ohio each year.

What is Duchenne Muscular Dystrophy (DMD)?

DMD is a hereditary progressive neuromuscular disease and one of the most severe forms of inherited muscular dystrophy, according to the Ohio Department of Health. An estimated 20,000 cases are diagnosed each year worldwide.

What are some symptoms of DMD?

Most symptoms of DMD appear between the ages of 2 and 3, according to Cleveland Clinic. This progressive disorder causes muscle weakness and degeneration, can affect walking around the age of 10, and can lead to death in individuals by their teens and twenties.

Can DMD be cured?

As of now, there is no cure for DMD. Yet, thanks to advancements in treatment, many patients live into their thirties and beyond, according to WebMD.

By screening for DMD shortly after birth with the Newborn Screening Program, parents can plan treatment options to help their child.

Dr. Jerry Mendell, a pediatric neurologist and principal investigator in the Center for Gene Therapy at Nationwide Children’s Hospital in Columbus, helped develop gene therapy that shows promise in treating DMD, the Dispatch previously reported.

Gene therapy is a medical approach that treats or prevents disease by correcting the underlying genetic problem, according to the National Library of Medicine.

This kind of therapy can be done in two ways:

  • Introduce a new gene into cells to help fight a disease

  • Introduce a non-faulty copy of a gene to stand in for the altered copy causing disease

Gene therapy can slow the progression of symptoms and improve the quality of life.

The newborn screening program on all newborns is required by Ohio law. According to information from the Office of the Governor, the screenings will help improve the health outcomes of nearly 300 babies each year.

This article originally appeared on Cincinnati Enquirer: Ohio first in US to screen newborns for DMD. Here's what to know

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