Category: Rare Disease

Five Strategies to Tailor Your Pediatric Clinical Trials to the Child’s Needs

Three Ways Patients and Advocates Can Inform Rare Disease Trials

PPD Heroes: Advocating for the Patient’s Voice During COVID-19

Cell and Gene Therapy Lesson 5: Lab Concierge – Extreme Focus on the Rare Disease Patient and Precious Study Sample Logistics Through an Innovative Lab Concierge Program

Rare Disease Research: The Benefit of Registries

What’s on the Horizon for Oncology?

Natural History Studies in Rare Diseases and Genetic Biomarkers

Patient Engagement in Clinical Trial Protocol Design and Recruitment Strategies

The Benefit of Registries for Real-World Evidence Generation in Rare Disease Research