Case Study
CureDuchenne LinkTM
The Next-Generation Data-Sharing Platform to Accelerate a Cure for Duchenne and Becker Muscular Dystrophy
BC Platforms is proud to partner with CureDuchenne to launch centralized Duchenne and Becker muscular dystrophy data hub, CureDuchenne Link™. This data hub helps to accelerate development of future treatments for this disease, by connecting data and biospecimens provided by the patient community with scientists and drug developers.
Background
CureDuchenne is a global leader and non-profit organization focused on finding and funding a cure for Duchenne muscular dystrophy (DMD), a progressive, muscle-wasting disease that affects more than 300,000 individuals worldwide. The disease is typically diagnosed around 5 years of age, leading to an inability to walk by age 12, and individuals rarely survive beyond their late twenties.
Challenge
To accelerate research to find a cure for this disease, CureDuchenne set up a biobank in 2019 to provide neuromuscular researchers with access to patient biospecimens. However, they soon realized that they needed a more scalable solution that could be launched nationwide, and which provided greater amounts of clinical detail alongside the biospecimens. Importantly, this solution had to be participant-centric and able to integrate diverse types of data in a single data warehouse.
Actions
After interviewing and vetting multiple vendors, the team finally arrived at a solution. BC Platforms was chosen as a key vendor for this 10-year project to build a data hub that could integrate a variety of data sources through its Discovery and Research Platform, and also provide tools for aggregate data management and data sharing.
Solution - CureDuchenne Link
CureDuchenne Link is a centralized data hub which combines clinical data, biosamples, and patient-reported data for individuals with Duchenne and Becker muscular dystrophy. CureDuchenne Link allows qualified researchers access to coded data and biosamples in one all-encompassing platform. Holistic data is curated using industry-standard terminology, allowing harmonization with other research data sets.
The data warehouse uses BC|INSIGHT, a HIPAA-compliant translational research platform, which allows for:
- Automated ingestion and harmonization of clinical information and multiple data types including genomics and other omics data.
- Researchers to interrogate and visualize data on this platform quickly using in-built tools for data curation and statistical analysis or customize their data exploration with the open analytics ecosystem.
- Fine-grained access management and auditing tools, ensuring that CureDuchenne can maintain data integrity and patient confidentiality while allowing for easy collaboration with researchers and drug developers.
Impact
Nationwide participant enrollment for CureDuchenne Link opened on July 9, 2021, and the team is looking to recruit 5,000 participants, including anyone who has been diagnosed with or is a carrier of Duchenne or Becker muscular dystrophy. This data hub will help to accelerate the discovery of biomarkers, as well as the development of future treatments for this disease.
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