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December 13, 2024

Real-world evidence in the cloud: Tutorial on developing an end-to-end data and analytics pipeline using Amazon Web Services resources

In the rapidly evolving landscape of healthcare and drug development, the ability to efficiently collect...

November 27, 2024

Autosomal Recessive Polycystic Kidney Disease (ARPKD) is a rare genetic disease. It occurs in about one in 20,000 births...

November 27, 2024

The mission of the Polycystic Kidney Disease Foundation is to fund research, advocate for patients, and ultimately to end PKD..

December 12, 2024

Item response theory (IRT) models are an increasingly popular method choice for...

November 27, 2024

Duchenne muscular dystrophy (DMD), a rare pediatric disease, presents numerous challenges when designing clinical trials...

December 1, 2024

Naomi Knoble, and Lindsey T. Murray on behalf of the Rare Disease Clinical Outcome Assessment Consortium. View all...

September 30, 2024

Type 1 diabetes (T1D) is an autoimmune disease characterized by the destruction...

October 8, 2024

Estelle Haenel, Celeste A. Elash, Katie Garner, Megan Turner, Scottie KernAbstract Electronic Clinical Outcome Assessment (eCOA) C

September 27, 2024

Purpose Determining if group-level differences in health outcomes are meaningful has recently been neglected...

August 1, 2024

It is estimated that more than 350 million people worldwide live with a rare disease.