Cure SMA Launches Spinal Muscular Atrophy Care Center Network

Oct. 5, 2018 -- Cure SMA, a national non-profit dedicated to the treatment and cure of spinal muscular atrophy (SMA), today announced the launch of the SMA Care Center Network, a national clinical data registry. The goal of the SMA Care Center Network, the first and only SMA-specific clinical data registry, is to develop an evidence-based standard of care that will improve the lives of all those affected by SMA.

The first phase will include 16 geographically diverse centers, with additional centers joining in subsequent years.

Spinal muscular atrophy 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. 

Following the first FDA approval of a treatment for SMA, and numerous other advances in SMA therapy development, the experience of SMA is changing rapidly. While SMA is currently the leading genetic cause of death for infants, these advances have offered new hope and a new challenge: to understand the changing landscape of SMA in order to deliver the best possible treatment and care to those affected by this rare genetic disorder.

“Good care requires time and coordinated effort by many specialists and the family. Improved care needs coordination among multiple centers to arrive at best practices and a standard of care,” said Kapil Arya, MD, of Arkansas Children’s Hospital in Little Rock, one of the first centers to join the network. Arya is also an assistant professor of Pediatrics in the University of Arkansas for Medical Sciences (UAMS) College of Medicine. “The SMA Care Center Network will drive research towards establishing such standards of care.”

In addition to Arkansas Children’s Hospital, the inaugural centers are Gillette Children’s Specialty Healthcare, St. Paul, MN; University of Rochester Medical Center, Rochester, NY; and the University of Utah Primary Children’s Hospital, Salt Lake City, UT.

 “As treatments extend lifespan, we will need more doctors and healthcare providers trained in SMA care. In addition, healthcare providers need to adapt quickly to the community’s changing needs,” said Mary Schroth, MD, chief medical officer for SMA. “The data collected in this registry will help answer questions about the impact of SMA and develop strategies for optimal care for those affected.”

The network will also help expand access to approved SMA treatments, increase the number of sites for SMA clinical trials, and help accelerate and enhance insurance coverage and reimbursement.

Over the course of the next three years, Cure SMA will invest $9.1 million in the development of the SMA Care Center Network.

About Cure SMA

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, we’ve directed and invested in comprehensive research that has shaped the scientific community’s understanding of SMA. We are currently on the verge of breakthroughs that will strengthen bodies, extend life, and lead to a cure.  We have deep expertise in every aspect of SMA—from the day-to-day realities to the nuances of care options—and until we have a cure, we’ll do everything we can to support and advocate for those affected by the disease. Learn more about how you can help us reach a treatment and cure at www.cureSMA.org.