The Patient Voice 2026
Why Does Publish or Perish Apply to Patient Advocate Groups?
Course Director: Jeanine R. Jarnes, PharmD, MSc, BCOP, BCPS
Monday, February 2, 2026
15:00 – 16:00 PST
1.00 Hour CE Session*
Overview: Rare diseases in general, and lysosomal diseases in specific, are constantly competing against common diseases for attention, resources, and funding, in every country around the world. Therapy development for lysosomal diseases is challenging for many reasons, including the inherently small populations, and challenges to pull together resources and collaboration. To this day, most lysosomal diseases do not have approved treatments.
In order to present a united approach, advocacy organizations need to bring together all of the stakeholders including patients, caregivers, clinicians, researchers, and pharmaceutical entities to provide published data to support lysosomal disease therapy development and approvals. This session will identify why it is important to have industry, clinician and advocates involved; the importance of peer-reviewed published data; describe several different approaches to publishing; and illustrate examples of the results and impact of publications. Representatives from leading lysosomal disease patient advocacy organizations will share their experiences and help provide attendees with practical steps to establish successful publication strategies.
Format/Agenda: Expert panel interviews and discussion with focused questions intended to address the stated issues. The session concluded with audience Q&A.
15:00 Welcome and Introduction of Faculty
15:05 Focused Interview Questions for Faculty with Panel Discussion
15:50 Open Audience Q&A
16:00 Adjourn
Course Director and Chair:
Jeanine Jarnes, PharmD, BCOP, BCPS
MSc Pharmacogenomics
Board Certified Pharmacotherapy Specialist
Board Certified Oncology Pharmacist
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
Minneapolis, Minnesota, United States
Faculty:
Alan Finglas
Founder & Research Manager
MSD Action Foundation / SavingDylan.com
Dublin, Ireland
Cara O’Neill, MD
Chief Science Officer
Cure Sanfilippo Foundation
Pediatrician
Columbia, South Carolina, United States
Jennifer Jackson, MS
President, International Society for Mannosidosis and Related Diseases (ISMRD)
Durham, North Carolina, United States
Rebekah Palmer
Co-Founder, Next Generation of Cystinosis
Duluth, Minnesota, United States
Target Audience: This activity has been designed to meet the educational needs of physicians, physician associates, nurses, genetic counselors, researchers, patient advocates, and other attendees at WORLDSymposium who care for and treat patients with lysosomal diseases.
Learning Objectives:
At the end of this session, participants will be able to:
- Identify specific examples of advocacy-led publications and explain how these efforts advanced knowledge and clinical recognition of rare and underrepresented medical conditions.
- Explain the role of HCPs in developing peer-reviewed publications in conjunction with advocacy groups to benefit all stakeholder groups and describe how these benefits advance patient care and medical progress.
- Demonstrate the value of the universal scientific publication structure and peer-review process in establishing credibility and shaping clinical and research practice.
- Describe collaborative approaches for advocacy groups, industry, academia, and healthcare providers to co-develop peer-reviewed literature, including clinical studies, treatment guidelines, and consensus white papers.
WORLDSymposium presented the 3rd Annual Patient Voice held on Monday, February 2, 2026 from 15:00 – 16:00 PST just prior to the 4th Annual WORLDSymposium Speed Mentoring Event and the opening event, “Be the Catalyst“.