The Patient Voice 2025

Inequities in Access to Diagnosis, Care and Clinical Trials in Lysosomal Diseases

Monday, February 3, 2025
15:00 – 16:00 PST

Overview
The Patient Voice 2025 was an 1-hour CE-session, held on Monday, Feb. 3, 2025 from 15:00 – 16:00 PST. This session is intended to discuss population inclusion and access to care and clinical trials regardless of diversity and socioeconomics, with a focus on improving patient care globally. The speakers included strived to recognize individual perspectives and struggles for families despite backgrounds, socioeconomic, and racial barriers, and identify realistic ways to improve opportunities for all patients.

Course Director
Jeanine R. Jarnes, PharmD, MSc, BCOP, BCPS
Assistant Professor, Department of Pediatrics
University of Minnesota Medical School
Pharmacotherapy for Inherited Metabolic Diseases
Advanced Therapies Department
College of Pharmacy, Experimental and Clinical Pharmacology
University of Minnesota
Minneapolis, MN, USA

Chair
Terri L. Klein
President and CEO
National MPS Society
Durham, North Carolina, USA

Faculty
Cyndi Frank 
Co-President, Co-Founder
Gaucher Community Alliance
Sonoma, California, USA

Jenifer Waldrop
Executive Director
Rare Disease Diversity Coalition (RDDC)
Denver, Colorado, USA

Agenda
15:00   Welcome & Introduction (Terri Klein, Chair)
15:05   A Patient’s Perspective  (Cyndi Frank)
15:20   The Inequities in the Rare Disease Community (Jenifer Waldrop)
15:35   From Awareness to Action: Equity in Rare Disease Outcomes (Terri Klein)
15:50   Panel Discussion and Audience Q&A
16:00   Adjourn