Leigh Syndrome Patient Registry Data AND CDISC STANDARDS

PRESS RELEASE

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Cure Mito Foundation and Sumptuous Data Sciences, LLC partner on alignment and interoperability of Leigh Syndrome Patient Registry Data with Regulatory Submission Standards

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Cure Mito Foundation (Cure Mito) and Sumptuous Data Sciences, LLC (Sumptuous Data Sciences) announce a partnership on alignment and interoperability of Leigh Syndrome Global Patient Registry Data, developed by Cure Mito Foundation with Regulatory Submission Standards.

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Cure Mito is a parent-led, volunteer foundation, dedicated to advancement of education and research for Leigh Syndrome and mitochondrial disease. Cure Mito started Leigh Syndrome (LS) Global Patient Registry in September, 2021 in order to identify and learn about LS patient population, facilitate clinical trial recruitment, bring together patients and researchers from around the world. The registry has over 180 enrolled patients from at least 25 countries as of May, 2022. The poster with initial results has already been published in February, 2022, and a paper is currently in progress.

However, establishment of interoperability of registry data with acceptable submission standards like Clinical Data Interchange Standards Consortium (CDISC) is essential in order to accelerate the development of therapies and is a critical milestone in case of rare diseases. CDISC Standards are required for regulatory submissions to FDA (U.S.) and PMDA (Japan).

 

Sumptuous Data Sciences is focused on providing biostatistics and data solutions services in healthcare sector with vision to work towards bridging the gap between healthcare and clinical data analysis and management. Sumptuous Data Sciences along with its Canada and India subsidiaries is working with pharma and biotech industry for development of data solutions and offers data operations services.

 

As a result of a collaboration, Cure Mito and Sumptuous Data Sciences will conduct a pilot project for establishment of such interoperability of Leigh Syndrome patient-populated registry data with CDISC standards. The result will be an overview of steps required to conduct such conversion and an assessment regarding conversion of patient-populated data to CDISC and its applicability to other mitochondrial and rare disease patient registries and natural history studies.

 

Pallavi Bakare, Director of Data Standards Governance and Implementation said: “Real World Data and Patient registries offer plenty of challenges during governance and compliance, analysis and reporting of data. We at Sumptuous; believe to make a difference with our expertise, to facilitate interoperability of this data with industry data standards like CDISC. This will help to accelerate development of therapies which is so critical in terms of Rare Diseases like Leigh Syndrome which do not have any known treatment yet.”  

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"We're excited to undertake this pilot project to see how patient reported data can be converted to ​meet the regulatory standards. ​We hope that what we can learn from this process will help provide a roadmap to inform and enhance research in other mitochondrial diseases. " - said Saima Kayani, MD, Child Neurologist with clinical focus on on Neurogenetics/Neurometabolic diseases and a scientific/medical advisor to Cure Mito foundation.

 

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Cure Mito Foundation is a parent-led 501(c)(3) nonprofit organization that was founded in 2018 as a Cure SURF1 Foundation by a group of families determined to fight for their children’s lives. In 2021, after successfully blazing the trail for SURF1 gene therapy at the University of Texas Southwestern Medical Center, the mission of the organization expanded and the name was updated.  Cure Mito’s primary focus is advancing research towards a cure for Leigh syndrome and eventually for Mitochondrial disease as a whole.  Cure Mito is a volunteer organization with 100% of funds used towards research. Patients are at the heart of everything Cure Mito does. For more information please visit: curemito.org.

 

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Sumptuous Data Sciences (SDS) is a specialized data solutions provider focused on biostatistics, clinical data operations and reporting functions. Sumptuous Data Sciences is a global services and solutions provider with operations in USA, Canada, and India. SDS operates in staffing, functional services, as well as project -based consulting services model. SDS is an ISO 9001 certified organization, has been member of CDISC, and has been active contributor in development of innovative processes and solutions through industry wide efforts.

 

Cure Mito contact: infor@curemito.org

Sumptous Data Sciences contact: pallavi.bakare@sumptuous-ds.com