Cure SMA Launches National Grassroots Advocacy Campaign for Spinal Muscular Atrophy Newborn Screening
By: Cure SMA
ORLANDO, Fla. - July 2, 2017 - PRLog -- Cure SMA, a leading national non-profit organization dedicated to achieving a world without spinal muscular atrophy (SMA), along with partners in the SMA Newborn Screening Coalition, today announced the launch of a new grassroots advocacy campaign to implement newborn screening requirements for SMA in every state.
The launch of the campaign was announced at the Annual SMA Conference in Orlando, FL – the largest SMA conference in the world. More than 2,200 families, researchers and clinicians attended the conference.
SMA is a neuromuscular disease and the number one genetic cause of death in children under two years old. The disease is caused by a mutation in the survival motor neuron 1 gene (SMN1). In a healthy person, this gene produces a protein that is critical to the proper 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.
"For many years, families whose children were diagnosed with SMA were told that there was no approved treatment and little that could be done to help them. After decades of work by our community, that has changed," said Kenneth Hobby, president of Cure SMA. "We now have an approved treatment, multiple other promising treatments being tested in trials, and strong clinical evidence that beginning treatment as early as possible is of the utmost importance. Newborn screening is the best opportunity we have to change the course of SMA for the next generation and beyond."
From June 29-July 2, the conference brought together leading scientists and doctors with those affected by the disease. As part of the event, SMA families were oriented to the campaign and how they can engage with their local leaders and advocate screening every newborn for SMA.
The campaign launch follows the passage by the Missouri House and Senate of a bill authorizing statewide screening for the disease. The legislation is currently awaiting Governor Eric Greitens signature. Representative Becky Ruth of Festus, Missouri sponsored the Missouri screening legislation and was on hand to share her experience with SMA advocates.
"As a grandmother who lost a grandson to a rare disease, I know how important it is to give these kids every chance to be happy and healthy, and potentially save their lives. I was proud to sponsor legislation in Missouri that will make us the first state to start screening for SMA. I can't wait to support and cheer on these families as they work to bring screening to their states too," said Ruth.
SMA advocates now return home across the country to encourage their leaders to enact newborn screening requirements in their states as well.
The campaign for SMA newborn screening follows the December 2016 FDA approval of Spinraza, the first approved treatment for SMA. In clinical trials of the drug, infants treated before showing symptoms gained more motor milestones than those treated after showing systems. The most severe forms of SMA cause rapid motor neuron loss, providing a small window for optimal treatment—an opportunity often lost due to delays in diagnosis that could be avoided through newborn screening.
While each state screens newborns according to its own standard list of tests, the states receive guidance from the federal Recommended Uniform Screening Panel (RUSP), administered by the Secretary's Advisory Committee on Heritable Disorders in Newborns and Children (SACHDNC), part of the U.S. Department of Health and Human Services. In May, the committee announced that it had accepted Cure SMA's nomination of SMA to the RUSP, where it is currently under review.
The intention is for the grassroots advocacy campaign to run alongside the RUSP evidence review process, maximizing the community's opportunity to move screening forward on both the state and federal levels. As part of this campaign, grassroots advocates in several states will focus not only on state-level advocacy, but also on advocating with federal legislators from their states, to ensure that appropriate funding is maintained for newborn screening.
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 and AveXis.
Learn more at www.cureSMA.org.
Page Updated Last on: Jul 02, 2017