Rare Diseases/Orphan Drugs Archives - Xtalks

Wednesday, August 12, 2026 | 11am EDT: Accelerate Cell Culture Media Development with Automated Cell Analysis

Running Paediatric Gene Therapy Studies: Operations and Oversight in Practice

September 09, 2026

Planning for Accelerated Approval: Timing is Everything

September 17, 2026

Access Program Process Changes to Increase Speed to Therapy

September 15, 2026

How to Use Your Existing Data to Predict Access Delays and Reduce Drop-Off

What TK2d Shows About Rethinking Rare Disease Trial Design

Strengthen Participant-Site Readiness for Complex Rare Disease Trials

How Genomic Data Improves Drug Development Timelines and Success

The Glomerular Revolution: Understanding the New Wave of Breakthroughs in Rare Kidney Diseases

Improve Recruitment Outcomes Through More Diverse Clinical Trials

3 Key Trends Transforming the Future of Rare Disease Clinical Development

February 27, 2026

When patients lead: Breaking barriers in ultra-rare disease drug development

Transforming Pediatric Rare Disease Trials Through Modeling and Simulation, and Site Networks

February 18, 2026

Site Selection Solutions Using Patient Insights for Rare Disease Trials

February 20, 2026

Responsible Data Return in Rare Disease Clinical Trials: What Sponsors Need to Know

February 26, 2026

Sanofi’s Cablivi Becomes First FDA-Approved Treatment for Pediatric aTTP

January 9, 2026

Waskyra Wins FDA Approval as First Gene Therapy for Wiskott-Aldrich Syndrome

December 12, 2025

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