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.
Day 1
Trial Design, Endpoints, Biomarkers & Safety
Regulatory Keynote Panel
What It Takes to Get It Approved: Navigating FDA Expectations in Lysosomal and Rare Disease Clinical Development
Session 1
Designing Trials That Succeed in Rare Diseases
Session 2
Developing Clinical Endpoints That Matter to Patients
Measuring meaningful benefit beyond biochemical correction
Session 3
Biomarkers and Long-Term Monitoring in Clinical Trials
Biomarkers as decision-support tools; distinguishing disease progression from treatment-related risk
Day 2
Therapeutic Modalities & Clinical Implementation
Session 5
Small Molecules, Chaperones & Substrate Reduction: Clinical Reality
Partial responders, CNS vs. peripheral benefit, and durability
Session 6
Enzyme, Biologic & Gene Therapies: Clinical Execution
Immunogenicity, residual disease, and real-world effectiveness
Session 7
Gene Therapy: Clinical Trial & Post-Trial Reality
Clinical management after dosing