11/7/26 From Parent Advocacy to IND: Building a Treatment Pathway for an Ultra-Rare Disease Previous Modeling Value Across the Rare Disease Patient Journey Next Commercial Success in Rare Disease Takes an Ecosystem: Aligning Manufacturers, Specialty Pharmacy, Providers, and Patient Support You Might Also Like From Awareness to Activation - Converting Rare Patient Identification into Trial Enrollment OPENING KEYNOTE - From Breakthrough to Access: How Advocacy and Policy Decide What Reaches Patients. Platform and Basket Trials in Rare Disease: What’s Working, What Isn’t, and What It Takes to Scale Pediatric Rare Disease Development: Evidence, Flexibility, and Regulatory Pathways Myth-Busting Hybrid & Home-Based Trials: What’s Real, What’s Not and What Scales
11/7/26 From Parent Advocacy to IND: Building a Treatment Pathway for an Ultra-Rare Disease Previous Modeling Value Across the Rare Disease Patient Journey Next Commercial Success in Rare Disease Takes an Ecosystem: Aligning Manufacturers, Specialty Pharmacy, Providers, and Patient Support You Might Also Like From Awareness to Activation - Converting Rare Patient Identification into Trial Enrollment OPENING KEYNOTE - From Breakthrough to Access: How Advocacy and Policy Decide What Reaches Patients. Platform and Basket Trials in Rare Disease: What’s Working, What Isn’t, and What It Takes to Scale Pediatric Rare Disease Development: Evidence, Flexibility, and Regulatory Pathways Myth-Busting Hybrid & Home-Based Trials: What’s Real, What’s Not and What Scales
