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December 12, 2024

The Power of Data Sharing and Collaboration Across LGMDs in a Pre-competitive, Neutral Environment

This event featured Dr. Volker Straub, a renowned neuromuscular genetics expert...

November 27, 2024

Voice of the Patient report Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for ARPKD Patients and their Families

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

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

Investigating item response theory model performance in the context of evaluating clinical outcome assessments in clinical trials

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

December 10, 2024

Biomarkers 101: A Patient’s Perspective on the Importance of Identifying New Biomarkers

Biomarkers are emerging as powerful tools in the field of kidney health, playing a crucial role in the diagnosis, monitoring...

November 27, 2024

A computational tool to optimize clinical trial parameter selection in Duchenne muscular dystrophy: A practical guide and case studies

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

December 1, 2024

Advancing rare disease measurement through the Rare Disease Clinical Outcome Assessment Consortium

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

November 27, 2024

C-Path’s Romero, Swingle Named to 100 Most Innovative and Influential Healthcare Leaders in AZ Big Media

C-Path CEO Klaus Romero and President & COO Kristen Swingle, President and COO were recently named to the 100 Most...

November 19, 2024

C-Path’s TRxA Announces $1 Million Award for Drug Development Project in Type 1 Diabetes

C-Path today announced that its Translational Therapeutics Accelerator (TRxA) program, in partnership...

November 17, 2024

Advancing Neonatal Drug Development: Celebrating Progress on World Prematurity Day

On this occasion of World Prematurity Awareness Day, we reflect on the immense progress we have made together and recognize...

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