Cure SMA and Parent Project Muscular Dystrophy Announce Collaboration

By Cure SMA | Published On March 19, 2019

Cure SMA and Parent Project Muscular Dystrophy Announce Collaboration

Today, Cure SMA and Parent Project Muscular Dystrophy (PPMD) announced their strategic collaboration to collect real-world data to improve the lives of those affected by spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (Duchenne). Both prominent organizations have joined forces with Prometheus Research as their principal informatics partner, enabling unprecedented levels of 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 quickly grown to 10 geographically diverse clinical sites nationwide. We expect that total to more than double by the end of 2019.

The primary goals of the SMA Care Center Network are to understand how life-changing 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.

Established in 2007, PPMD’s Duchenne Registry is the largest, most comprehensive patient-reported registry for Duchenne and Becker muscular dystrophy. Thanks to the participation of nearly 5,000 registrants, The Duchenne Registry recently published a 10-year report to inform researchers and sponsors on patient-reported outcomes in order to advance care and treatments for individuals with Duchenne. Similar to SMA, PPMD’s registry works with some 25 clinics through their Certified Duchenne Care Center program.

With both Cure SMA and PPMD playing a central and integral role in advancing treatment, the organizations are now collecting real-world evidence to further support the patient and research communities.

To do this important work, both Cure SMA and PPMD have partnered with Prometheus Research, implementing an innovative registry model, RexRegistry. This model allows data to be captured through electronic health records (EHR), and limits the data entry burden on providers or their support staff. With multiple disease communities now using the same underlying system, sites of care can more effectively and efficiently participate in these registries.

In the rapidly changing world of rare disease, Cure SMA and PPMD are steadfastly committed to shared learnings and working to develop greater collaborations across and within the rare disease space.

“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.”

“Since a small group of parents and grandparents formed PPMD almost 25 years ago, we have believed in the importance of innovation and patient data in the fight to end Duchenne," said Pat Furlong, Founding President & CEO of PPMD. “We are thrilled to collaborate with Cure SMA, working side by side to seize the power of patient data to end these devastating diseases,” she continued.

In December 2016, the FDA approved Spinraza as the first-ever approved therapy targeted at the underlying cause of SMA. The FDA is also reviewing Zolgensma. Zolgensma is a gene therapy for infants affected by SMA type 1, the most severe and most common form of the disease. A decision is expected in May. “With a second treatment being reviewed by the FDA and another six in clinical trials, we know this is just the beginning of our efforts to change the lives of those affected by SMA,” said Mary Schroth, MD, chief medical officer for Cure SMA. “The SMA Care Center Network is one of the most important tools to this mission.”

Topics: Support & Care, Our Impact, Front Page News

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