Hope for PDCD and Cure Mito Foundation unveil joint patient registry

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Cure Mito Foundation and Hope for PDCD Foundation, both patient-led foundations focused on advancing research and supporting families affected by Leigh syndrome and Pyruvate Dehydrogenase Complex Deficiency (PDCD), respectively, are excited to announce a launch of a PDCD patient registry. This innovative registry will be led by the Hope for PDCD foundation and hosted on the same registry platform as the well-established Leigh Syndrome patient registry, developed by the Cure Mito Foundation. This strategic move is aimed at enhancing patient convenience and improving data alignment and research effectiveness.

Both Leigh syndrome and PDCD are types of primary mitochondrial diseases mainly affecting children. There is an overlap between two conditions, with around 25-35% of PDCD patients also having Leigh syndrome, according to published literature.

Both registries will be hosted on the Sanford CoRDS registry platform. Sanford CoRDS is the largest, free international rare disease registry. It works to connect participants, advocacy groups and researchers in the field.

By hosting both registries on the same platform, we are reducing the burden on patients and caregivers. Now, individuals affected by either or both conditions can register and answer many questions just once, streamlining their participation in crucial research efforts. This integration not only eases the process for patients and families but also allows for better alignment and comparison of data across different but potentially related conditions. This will help accelerate research and ultimately improve outcomes for patients.

Frances Muenzer Pimentel, founder of the Hope for PDCD foundation and mom to Violet, who has PDCD, said: "Hope for PDCD is thrilled to launch the first ever international and patient initiated registry for PDCD on the CoRDS platform in conjunction with Cure Mito. Launching on CoRDS with Cure Mito allows the 25-35% of PDCD patients that also have a Leigh Syndrome diagnosis to complete both registries on one platform. This registry will become a powerful tool to better understand the disease and to help support the development of treatments and participation in clinical trials. The registry also signals to industry and academia that we are an organized and unified patient population in urgent need of a standard of care and effective treatments."

Patient registries are valuable sources of information not only for physicians, researchers, and families, but also for industry partners who are working to better understand the complexities of rare diseases. We are pleased that Hope for PDCD is working with Cure Mito to create a registry with the goal to advance treatments, optimize care, and ultimately improve the lives of those with PDCD." 

Sue Clement, Senior Director at Saol Therapeutics

Sophia Zilber, board member and patient registry director of the Cure Mito Foundation, said, "The concept of merging data from various registries is frequently discussed within the rare disease community. However, this is a complex task that requires a foundational alignment of data collection methods right from the start. Our collaboration is pivotal in facilitating this alignment. Since the inception of our Leigh syndrome patient registry two years ago, we have witnessed remarkable advancements and insights. With this collaborative effort, we are enthusiastic about replicating this success within the PDCD community and across our broader community."

The unified registry platform will facilitate deeper insights into PDCD and Leigh Syndrome, potentially unveiling commonalities that could lead to more effective treatments. It also opens avenues for patients to participate in a broader range of studies and trials relevant to their conditions.

Patients and caregivers interested in joining the registry can expect a user-friendly interface, robust data security, and the opportunity to contribute directly to the advancement of medical research. By participating, they become vital partners in the quest to understand and treat these complex diseases.

For more information about the PDCD patient registry, Leigh Syndrome patient registry, or to register, please contact [email protected] and [email protected].

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