PPMD is thrilled to announce our strategic collaboration with Cure SMA, one of the world’s leading rare disease patient advocacy organizations. Like PPMD, Cure SMA is looking to advance data collection and analysis on their patient population. Today, both organizations announced a collaboration with Prometheus Research to further their data collection and analysis goals. 

PPMD’s Duchenne Registry was established in 2007 and because of the support and participation of families like you, it is the largest, most comprehensive patient-reported registry for Duchenne and Becker muscular dystrophy. With nearly 5,000 registrants worldwide, the 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. The Duchenne Registry works with PPMD’s 24 Certified Duchenne Care Centers throughout the US.

With a consistent focus on advocacy to advance the development of therapies, both Cure SMA and PPMD have played a central and integral role in advancing treatments, and now add to their missions the collection of 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. The Prometheus Research data solutions have been designed to integrate and repurpose data from multiple sources to help patient groups, clinical researchers, and industry sponsors address common challenges in rare disease research.

Enhancing Duchenne Data Collection & Analysis

The major goal of The Duchenne Registry is to better understand Duchenne and the disease progression, collecting data directly from patients and caregivers. In Duchenne, the Prometheus Research model will combine data from multiple sources, including the patient-reported data in the PPMD Duchenne Registry AND clinician-reported data in electronic health records (EHR) at various hospitals across the United States to strengthen the data set and potentially answer additional questions about Duchenne. Cure SMA and PPMD are using the same technology partner in order to accomplish our data collection and analysis goals, the hope is to also reduce burden of data entry at clinics. With multiple disease communities now using the same underlying system, hospitals and clinics can more effectively and efficiently participate in these registries.

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. It is an amazing moment in the evolution of our organization to collaborate with Cure SMA and Prometheus Research, all 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.

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.

JOIN THE DUCHENNE REGISTRY

If you haven’t joined The Duchenne Registry or haven’t updated your profile recently, please do so today! Become a citizen scientist and share your data so you can further the power of the Registry!