Cure SMA Launches Patient Registry, Care Network with Aim of Improving Treatment and Access

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by Mary Chapman |

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brain-derived neurotrophic factor, SBMA

Aiming to establish a standard of care for people affected by spinal muscular atrophy (SMA), Cure SMA has unveiled what’s being touted as the first national clinical data registry specific to this neuromuscular disease.

Cure SMA will invest $9.1 million over three years in the registry, part of a new SMA Care Center Network, according to a press release. Initially consisting of 16 centers throughout the United States, this network will also help increase access to treatments and the number of clinical trial sites, and to improve insurance coverage and reimbursement.

The first four centers are Arkansas Children’s Hospital, Little Rock, Arkansas; Gillette Children’s Specialty Healthcare, St. Paul, Minnesota; University of Rochester Medical Center, Rochester, New York; and the University of Utah Primary Children’s Hospital, Salt Lake City, Utah. The other 12 will be announced later this year.

Care centers are expected to continue to be added to the network.

Patient care data gleaned at these centers will add to what’s known about the impact of SMA, and help formulate the best ways of treating SMA patients.

Together, such efforts can move care toward a more cutting-edge standard, and promote best practices among healthcare providers nationwide. Because data will better reveal where communities of patients are, the registry may also lead to better treatment access.

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

Cure SMA’s multifaceted approach to care mirrors the complex needs of SMA patients, the organization said. Those living with the disease are treated by a variety of medical specialists, including neurologists, pulmonologists, occupational and physical therapists, nutritionists, and orthopedists.

As SMA patients live longer due to the availability of approved treatment — and with promising therapies looming — their numbers will increase. New treatments will also affect patients in ways unfamiliar to some healthcare professionals. Those factors call for more SMA-trained providers who can adapt to a shifting SMA landscape, including older patients.

“Good care requires time and coordinated effort by many specialists and the family,” said Kapil Arya of Arkansas Children’s Hospital, and 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 toward establishing such standards of care.”

Other new SMA care efforts include the SMA Clinical Care Meeting, held as part of the annual SMA Conference; Cure SMA care series booklets; and physical and occupational therapy mentoring programs.