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Debra Miller first noticed that something was wrong when her four-year-old son Hawken struggled to keep up with other kids while playing soccer. After two long years of hospital visits and inconclusive tests, they were finally diagnosed correctly with Duchenne muscular dystrophy, a genetic condition characterized by progressive muscle degeneration and weakness that primarily affects boys. This inspired Debra and her husband Paul to found CureDuchenne, a non-profit organization aimed at accelerating the development of drugs for the treatment of Duchenne, in an attempt to save her son and thousands of other boys with this rare disease.
Over the past 18 years, CureDuchenne has grown into a leading advocacy organization serving the needs of the Duchenne community. Their innovative model of venture philanthropy has been extremely effective in funding groundbreaking research, early diagnosis and access to treatment. While hiring drug developers working on this disease, the CureDuchenne team encountered a recurring problem: Scientists were not able to easily access biological samples from patients to advance their research. Although some academic institutions had individual collections, these samples were often not associated with the appropriate medical records and were often in silos, making it difficult for researchers to use.
To meet this challenge, CureDuchenne set up a pilot biobank in 2019, but soon realized that they needed a more scalable solution that could be rolled out nationwide. Most importantly, the platform needed to be participant-centric and able to integrate various types of data into a single data warehouse. After interviewing and vetting several vendors, the team finally managed to find a solution. In the summer of 2021, CureDuchenne launched CureDuchenne Link ™, the first national Duchenne and Becker muscular dystrophy data center that directly links data and biological samples provided by the patient community to scientists and drug developers across the country. whole world.
CureDuchenne Link is powered by technology from BC Platforms, a global leader in healthcare data management, analysis and access. The data warehouse uses BC | INSIGHT, a HIPAA-compliant translational research platform, which enables the automated ingestion and harmonization of clinical information and multiple types of data, including genomics and others. omics data. Researchers can quickly query and visualize data on this platform using built-in tools for data curation and statistical analysis or customize their data exploration with the open analytics ecosystem. Additionally, BC | INSIGHT provides accurate access management and auditing tools, ensuring that CureDuchenne can maintain data integrity and patient privacy while enabling easy collaboration with researchers and drug developers.
“Choosing the BC platforms gave us the most power and flexibility,” says Debra Miller. “We have put so much energy and effort into building this platform that we would love nothing more than to be able to shorten this process for other rare disease organizations looking for similar solutions. BC Platforms is a key technology partner that we recommend.
The registration of participants nationwide for CureDuchenne Link opened on July 9, 2021. The team is seeking to recruit 5,000 participants diagnosed or carrying Duchenne or Becker’s muscular dystrophy. Ultimately, this data center, providing a holistic view of biological samples integrated with clinical, genomic and patient-reported data, will be an invaluable resource for researchers and drug developers. This will undoubtedly help accelerate the discovery of biomarkers, as well as the development of future treatments for this disease.
Learn more about BC Platforms solutions and how they can meet all of your research needs. www.bcplatforms.com/pharma-biotech