Parent Project Muscular Dystrophy's Duchenne Patient Registry Reaches 10-Year Milestone

By Staff, Parent Herald May 04, 07:16 am
In World's Largest Duchenne Registry, Patients Contribute Data Used by Industry, Academic Researchers, and Clinicians
(Photo : Fred Dufour/AFP | Getty Images)

 Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced that The Duchenne Registry has reached a significant milestone collecting 10-years of patient-reported data. Formerly known as DuchenneConnect, The Duchenne Registry is widely considered the go-to patient registry for researchers and clinical investigators in the Duchenne space. With two drug approvals in the U.S. for Duchenne, The Duchenne Registry continues to provide important patient-entered data that informs clinical trial protocol and helps inform regulatory agencies.

To date, The Duchenne Registry has 4,200 registrants. This includes people living with Duchenne and Becker muscular dystrophy, as well as female carriers. Over 100 countries are represented. As a member of the TREAT-NMD International Neuromuscular Network, The Duchenne Registry shares several core data fields with the global Duchenne registry

According to PPMD Founding President and CEO, Pat Furlong, "Families affected by Duchenne and Becker muscular dystrophy do not always receive a proper diagnosis; fewer receive the most up-to-date treatments for these diseases. The Duchenne Registry has provided families access to top researchers and clinicians in the field, as well as notification about clinical trials that may help their child. Gathering the community into a registry with global partnerships provides crucial information to researchers and clinical investigators, and we believe has helped accelerate therapeutic discoveries. To see the seed of an idea from a group of passionate leaders in the Duchenne community blossom into a thriving online resource is truly phenomenal."

The Duchenne Registry currently has 17 industry partners and has helped recruit for approximately 60 clinical trials. Data from the Registry has been used in 7 publications and an additional 3 publications are pending. In addition, The Duchenne Registry was honored to be selected as one of the Patient-Powered Research Networks (PPRNs) in PCORnet, the National Patient-Centered Clinical Research Network. The Duchenne Registry has been a PPRN within PCORnet for both Phase 1 and 2 of PCORnet, from 2014 - 2018.

Not only has data from The Duchenne Registry been used to improve care and enhance the development of potential therapies for Duchenne, but the experiences families share through this registry inform regulators. As more therapies advance in the clinical trial pipeline and interactions between the Duchenne community and federal regulatory agencies like the FDA increase, the importance of patient-reported data has never been more critical for decision makers.

New Name, New Features

To mark the 10th anniversary of The Duchenne Registry, PPMD has implemented new features and tools to help enhance the user experience.

New features include a more modern, streamlined homepage and website that is more fully integrated with the acclaimed PPMD patient advocacy website. In addition, once registrants log in to their registry account, they will experience a new, more interactive registry dashboard. A new feature on the dashboard is the medications module which will be critical in this new era of approved Duchenne therapies.

Participants in The Duchenne Registry will still find the same medical surveys to complete, and the same ability to upload medical records, add healthcare providers, track height and weight, and add family members. Registrants will also continue to have direct access to PPMD's board-certified genetic counselors who specialize in Duchenne.                          

As always, information entered into The Duchenne Registry is kept confidential and secure.  Invitae, the company hosting the registry platform, complies with important research and privacy regulations for protecting patient data.  However, de-identified data is shared with the community, including registrants, researchers, and clinicians.

What's Next?

The Duchenne Registry will continue to improve the user experience over the coming months.

New functionality planned for the Registry includes upgrading the "View Data" feature so registrants can view their data in a graphic-rich format and see how they compare to others in the community. PPMD greatly appreciates the time and effort spent by families inputting and updating their data in the Registry, and a new section on the Registry dashboard ("Your Data at Work") highlights the impact of this data. Efforts will also be made to make the inputting of data less burdensome to families.    

In a post-approval landscape, The Duchenne Registry will also make several enhancements to the data collected so that industry partners can improve post-marketing surveillance of their products.

The History of the Registry

The Duchenne Registry (formerly DuchenneConnect) began in 2007, when a group of thought leaders in the Duchenne and Becker muscular dystrophy community began discussing the need for a new kind of resource that would connect and serve the needs of the entire community. What they envisioned was a central hub that would bring together those living with Duchenne or Becker, along with their families and caregivers, to connect them with medical research, clinical care, clinical trials, and each other. At the same time, it would also be a resource for researchers and industries with an interest in Duchenne, allowing access to aggregate, de-identified data provided by patients and their families - information that could prove vital to advances in care and treatment. Today, the result of this endeavor is The Duchenne Registry, the largest, most comprehensive registry for Duchenne and Becker muscular dystrophy, as well as female carriers.

Ann Martin, Director of The Duchenne Registry, adds that the success of this platform is a direct result of community participation: "When families join and update their account in The Duchenne Registry, they are strengthening the power of a 10-year-old network of patient-powered data that will be used to improve care for people living with Duchenne and increase understanding of the disorder. Families become citizen scientists by contributing to real scientific research, without ever leaving their homes."

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