GRIDS 2026 Symposium
Program Agenda
Advancing Therapeutic Development and Clinical Trials
in Lysosomal & Rare Disorders
Tentative program — subject to change. Final schedule with speakers and times coming soon.
Trial Design, Endpoints, Biomarkers & Safety
Session 1
Designing Trials That Succeed in Rare Diseases
Session 2
Developing Clinical Endpoints That Matter to Patients
Session 3
Biomarkers in Clinical Trials: Utility, Limits & Misuse — Biomarkers as Decision-Support Tools
Session 4
Safety, Tolerability & Long-Term Monitoring of Rare Diseases During Trials
Therapeutic Modalities & Clinical Implementation
Session 5
Small Molecules, Chaperones & Substrate Reduction: Clinical Reality
Session 6
Enzyme, Biologic & Protein Therapies: Clinical Execution — Immunogenicity, Switching Therapies, and Real-World Effectiveness
Session 7