Indiana Becomes Fourth State to Adopt Newborn Screening for Spinal Muscular Atrophy
Legislation will help promote life-saving treatment of the leading genetic cause of death for infants under two years of age.
By: Cure SMA
CARMEL, Ind. - May 11, 2018 - PRLog -- Governor Eric Holcomb today will hold a signing ceremony for Indiana House Bill 1017, adopting newborn screening for spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID).
"This new law will help more children live longer and better lives," Gov. Holcomb said. "Working together, our state and community leaders can continue to make strides in reducing infant mortality and improving quality of life for all Hoosiers."
Dubbed "Graham's Bill" in honor of Graham Vollmer, the bill was largely advanced by the efforts of Adrienne Vollmer, Graham's mother, and State Representative Doug Gutwein, the bill's lead sponsor. The signing will be held at the Cure SMA Walk for Graham, an event organized by the Vollmer family and benefitting Cure SMA, a national non-profit organization that supports research and advocacy for SMA.
"Our family has been so honored to be a part of the process to add SMA to Indiana's newborn screening. We hope this legislation will be able to save families from some of the heartache we experienced when Graham was diagnosed. Our Walk for Graham this year is the perfect way to celebrate the success of this bill and the families it will impact," said Vollmer.
Spinal muscular atrophy (SMA) is a neuromuscular disease caused by a mutation in the gene survival motor neuron 1. In a healthy person, this gene produces a protein that is critical to the function of the nerves that control muscles. Without it, those nerve cells cannot properly function and eventually die, leading to debilitating and often fatal muscle weakness. On average in the US, a baby with SMA is born each day.
In clinical trials for Spinraza, which was approved by the FDA in December 2016, infants treated before showing symptoms gained more motor milestones than those treated after showing symptoms. The most severe forms of SMA cause rapid motor neuron loss, providing a small window for optimal treatment—an opportunity often lost due to the diagnostic delays that many families experience.
"Indiana is at the leading edge of an effort to save the lives of babies born with spinal muscular atrophy. SMA is currently the number one genetic cause of death for infants under two years of age, but we now have an historic opportunity to change that through newborn screening and early treatment," said Kenneth Hobby, president of Cure SMA. "We congratulate SMA advocate Adrienne Vollmer, Representative Doug Gutwein, and Governor Eric Holcomb on their quick action on this issue, and hope that more states will follow their lead in the coming months."
In February, the federal Advisory Committee on Heritable Disorders in Newborns and Children recommended that screening for SMA be implemented nationwide. This recommendation is currently awaiting final approval by Health and Human Services Secretary Alex Azar. Though the federal recommendation is critical, the decision to implement this recommendation ultimately rests with each individual state.
About Cure SMA and the SMA Newborn Screening Coalition
Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA)—a disease that takes away a person's ability to walk, eat, or breathe. It is the number one genetic cause of death for infants.
Since 1984, Cure SMA has directed and invested in comprehensive research that has shaped the scientific community's understanding of SMA. Cure SMA has deep expertise in every aspect of SMA—from the day-to-day realities to the nuances of care options—and administers a wide-ranging roster of programs to support children and families affected by the disease. This includes the Annual SMA Conference, held each year for families affected by the disease, and for researchers and clinicians involved in treatment and care for SMA.
In addition to staff members from Cure SMA, the SMA Newborn Screening Coalition includes representatives from pharmaceutical companies Biogen, AveXis and Genentech/Roche.
Learn more at www.cureSMA.org.
Page Updated Last on: May 15, 2018