Editor’s note: This op-ed was originally published in The Wall Street Journal on Sept. 14, 2025.
What if, for $8 per newborn, America could save hundreds of boys a year from wheelchair-dependent lives that end many decades too soon? After public comment closes on Sept. 15, the Department of Health and Human Services will review whether to include Duchenne muscular dystrophy as a part of the Recommended Uniform Screening Panel — the list of conditions included in routine newborn screenings across the country.
My son Eliot was diagnosed with DMD at 7 months old. He was screened fortuitously after a routine blood test during a hospitalization for an unrelated viral infection. An abnormal result led his pediatrician to investigate, and as a result of his early diagnosis, Eliot received Elevidys, a gene therapy under the Food and Drug Administration’s “accelerated approval” pathway. These days, Eliot jumps, runs and climbs like an unaffected kindergartner — so much so that I, not the disease, am the one putting limits on his activity.
Most boys with DMD are diagnosed at 4 to 5 years old when they start showing gross motor skill delays, difficulty getting up, and falling. (While in rare cases girls can present with symptoms, they are almost always only carriers.) On average, there is a critical 2 1/2-year delay between initial symptoms and diagnosis. The problem with a delayed diagnosis is that Elevidys doesn’t work as well on older patients. Critics who claim Elevidys is ineffective are wrongly blaming the therapy for the irreversible disease progression seen in older boys.
According to the Parents’ Project on Muscular Dystrophy, approximately $8 per infant tested would cover the cost to screen all newborns for DMD. It would also cover the genetic testing for the approximately 350 boys each year who will show elevated creatine kinase levels — usually the first sign of the disease.
The current screening list includes spinal muscular atrophy, sickle-cell disease and cystic fibrosis, along with other metabolic and endocrine diseases. Interventions for many of these conditions now exist. Although not all listed diseases have cures, newborn screening offers critical data on their prevalence, encouraging medical researchers and private biotech companies to create treatments where there is the greatest need.
Early knowledge of the disease and subsequent early intervention is key to changing DMD from a death sentence to a potentially manageable disease. Clinical trials have shown that boys who received the gene therapy at a young age fared better than placebo groups and external controls. Children 4 to 5 years old had better outcomes than the boys who received the treatment at an older age.
Elevidys does work, but as with other treatments — from chemotherapy for cancer to Trikafta for cystic fibrosis — timing is everything. Adding Duchenne muscular dystrophy to the HHS newborn screening panel gives these boys a chance to live better and more functional lives.
