This past weekend, babies born throughout Nebraska were the first in the state to be screened for Spinal Muscular Atrophy (SMA), a genetic disorder that starts in the central nervous system and affects all the muscles in the body. SMA is one of the leading genetic causes of infant death, it affects roughly 1 in 11,000 live births and almost 1 in every 50 Americans is a carrier for this disease.
SMA was added to the panel of 32 different genetic and metabolic conditions of which newborns are screened following the passage of Legislative Bill 825, introduced by Senator Robert HIlkemann in 2020.
Hilkemann stated, “Prior to this weekend, it was a statistical certainty that a baby would be born with SMA and their parents wouldn’t know. They would not have the opportunity to seek the treatments that are available right now that could save their child’s life, but now they do.”
FDA approved treatments for SMA have proven successful with early detection and intervention. The positive outcomes in clinical trials led the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) to recommend to the US Secretary of Health and Human Services that SMA be added to the Recommended Uniform Screening Panel (RUSP) in 2018.
The Nebraska Newborn Screening Advisory Committee recommended in 2019 that SMA be added to the Nebraska panel.
“Newborn screening saves and improves lives by giving families with babies born with SMA the information they need to make timely decisions about life-saving treatment and care,” said Kenneth Hobby, President of Cure SMA, the leading national organization representing individuals with SMA and their families. “On behalf of Nebraska residents with SMA and their families, Cure SMA thanks Hilkemann for authoring the law to add SMA to the state’s newborn screening panel and the Nebraska Department of Health and Human Services for implementing the law to ensure Nebraska babies are screened for SMA, the leading genetic cause of death in U.S. infants.”