Rare Daily Staff

Cure SMA and Parent Project Muscular Dystrophy said they have entered into a strategic collaboration to collect real-world data to improve the lives of people affected by the genetic neuromuscular diseases spinal muscular atrophy and Duchenne muscular dystrophy.

Both organizations have joined forces with Prometheus Research as their principal informatics partner, enabling collaboration on data and technology.

The Cure SMA clinical data registry contains patient data submitted by partner care centers. These partner care centers form the Cure SMA Care Center Network. The SMA Care Center Network and clinical data registry launched in October 2018 and has grown to 10 geographically diverse clinical sites nationwide. It expects to double the number of sites by the end of 2019.

The primary goals of the SMA Care Center Network are to understand how therapies are transforming the experience of SMA, and to identify an evidence-based standard of care that will allow individuals to receive maximum benefit from these therapies.

 “With new treatments, the experience of SMA is rapidly shifting, and healthcare providers need to adapt quickly to these changing needs,” said Kenneth Hobby, president of Cure SMA. “The goal of the SMA Care Center Network is to develop an evidence-based standard of care that will address this changing landscape, expand access to approved SMA treatments, and help accelerate and enhance insurance coverage and reimbursement.”

PPMD’s Duchenne Registry, established in 2007, is the largest, most comprehensive patient-reported registry for Duchenne and Becker muscular dystrophy. The registry boasts 5,000 registrants. The organization recently published a 10-year report on to inform researchers and sponsors on patient-reported outcomes in order to advance care and treatments for individuals with Duchenne.

Similar to the Cure SMA, PPMD’s registry works with some 25 clinics through their Certified Duchenne Care Center program. The two organizations are now collecting real-world evidence to further support the patient and research communities.

Under their partnership with Prometheus Research, the organizations are implementing an innovative registry model, RexRegistry. This will allow data to be captured through electronic health records and limit the data entry burden on providers or their support staff.