HACKENSACK, N.J., March 19, 2019 /PRNewswire/ -- Today, Cure SMA and Parent Project Muscular Dystrophy (PPMD), two of the world's leading rare disease patient advocacy organizations, announced their strategic collaboration to advance clinical data aggregation and reaffirmed their commitment to improving health outcomes for 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 collects electronic health record based patient data from a growing set of sites in the newly established Cure SMA Care Center Network; this data is being used to facilitate a wide range of clinical research and quality improvement initiatives. Launched in late-2018, the SMA Care Center Network has quickly grown to include ten geographically diverse clinical sites nationwide, with plans to add nationwide newborn screening and more than double in size by the end of 2019. The primary goals of the SMA Care Center Network are to understand how life-changing SMA 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 and including patient-reported data from nearly 5,000 registrants, PPMD's existing Duchenne Registry is the largest, most comprehensive patient-reported registry for Duchenne and Becker muscular dystrophy in the world. Data from The Duchenne Registry's first decade of success was recently published in a 10-year report to inform clinical researchers and industry sponsors on patient-reported outcomes. PPMD is now partnering with Prometheus on a next-generation data infrastructure capable of enhancing these patient-reports with real world data from clinical systems (including electronic health records), administrative claims, and social determinants. Like SMA, PPMD's registry will integrate with dozens of specialty clinics through their well-established Certified Duchenne Care Center program.

The Prometheus data solutions for both Cure SMA and PPMD have been designed to integrate and repurpose data from disparate health sources to help clinical researchers and industry sponsors address common challenges, such as monitoring the changing natural history of each disease and the outcomes these patients experience over longer periods of time than traditional studies typically examine. Underpinning these capabilities are Prometheus' nearly 20 years of clinical research informatics expertise and RexRegistry, the industry's first agile data hub that creates research-grade data assets capable of minimizing burden on data partners while dynamically supporting new combinations of data sources, data uses, and stakeholders over time. With both organizations now using the same underlying system, care sites can more effectively and efficiently participate in these initiatives.

"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-reported outcomes and electronic health records in order to further our missions to end these devastating diseases," she continued.

"This is a very exciting time in human health, and for rare disease advocacy specifically," added David Voccola, cofounder of Prometheus Research. "The advent of modern data standards is unlocking potentially transformative collaboration models. Prometheus is thrilled to be supporting these two remarkable organizations as they lead the way to this promising new future."

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

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 and follow Cure SMA on Facebook, Twitter, Instagram, and YouTube.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives. We will not rest until every person has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube. 

About Prometheus Research

Prometheus Research is the principal informatics partner to renowned health organizations engaged in ambitious translational research endeavors and quality improvement initiatives. Our agile data platform, RexRegistry, empowers our partners to acquire, integrate, and repurpose disparate sources of health data to accelerate scientific discovery and patient access to new therapies. Together with our consulting services, Prometheus helps forward-thinking institutions build data assets today that will continue advancing the state of care for generations to come.

SOURCE Parent Project Muscular Dystrophy

Related Links

http://www.parentprojectmd.org