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November 27, 2024

Autosomal Recessive Polycystic Kidney Disease (ARPKD)Adjunct EL-PFDD Scientific Workshop Report

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 authors and aff

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.

July 29, 2024

C-Path and experienced private sector partners collaborated with global health organizations to create didactic video materials...

June 12, 2024

Digital measures may provide objective, sensitive, real-world measures of disease progression in Parkinson’s disease (PD).