Chris Arnold told a group of Nebraska legislators a story Wednesday.
He told members of the Legislature’s Health and Human Services Committee how he and his wife, Paula Lavigne, brought home their firstborn son, Wyatt, in July 2010 believing they had a perfectly healthy baby.
But Wyatt didn’t meet developmental milestones. That October, the Omaha couple learned that Wyatt had a genetic condition that meant they would lose him. And they did, when he was days past 5 months old.
Arnold told Wyatt’s story in support of Legislative Bill 825, a measure that would add that genetic condition, spinal muscular atrophy or SMA, to the state’s newborn screening panel, the heel-prick blood test all Nebraska babies are given at birth.
The panel now screens for 32 conditions that share a common feature: If caught early, babies can be treated in time to prevent or reduce the problems associated with them.
When Wyatt was born, there was no treatment for SMA, a progressive condition that leads to weakened muscles. In its worst forms, including the one Wyatt had, it affects those needed for vital functions like breathing and swallowing.
But new treatments, including a gene therapy the Food and Drug Administration approved in May, are making a difference in the lives of children with the condition. Arnold said he and his family have seen those improvements in children who’ve received them.
“Early diagnosis at birth is critical for Nebraska families diagnosed with SMA,” he said.
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Sen. Robert Hilkemann of Omaha said he hopes to get the bill approved this session so it can go into effect this summer. That would require the committee to send it on and the full Legislature to approve it. He brought the measure in partnership with Children’s Hospital & Medical Center.
“The longer we wait, it’s a statistical certainty that a baby or babies will be born with SMA and their parents won’t know,” he said. “And their parents won’t have the opportunity to seek the treatments that could save their child’s life.”
According to Cure SMA, an advocacy group, the condition, while rare, is the leading genetic, or inherited, killer of children younger than 2. On average, two babies are born in Nebraska each year with SMA. About 80 Nebraskans are living with the condition, and an estimated 38,500 residents carry the gene.
Dr. Geetanjali Rathore, Children’s interim division chief of neurology, said the condition causes irreversible damage even within the first days of life.
Early results indicate that very young children treated with the gene therapy were doing very well, developing almost normally.
“We have to give them that opportunity to live a full, healthy life and be able to participate in our community,” said Rathore, who has treated children with the new therapies.
Federal health officials added SMA to their list of recommended screenings for newborns in 2018. The Nebraska Newborn Screening Advisory Committee voted to recommend adding it to the state panel last year.
Adding the test would not add to the state’s costs, according to a review. Children’s officials estimated that adding the screen would boost the cost billed to insurance for the newborn panel by $5.50, bringing the total to $86.
Arnold said he and his family continue to tell Wyatt’s story because he didn’t get the chance to write it himself.
“Today,” he said, “I tell Wyatt’s story so children born with SMA can tell their own stories.”
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