WORLDSymposium™ 2026 Preliminary Program* on Lysosomal Diseases On Monday afternoon, WORLDSymposium presented an exciting program, Patient Voice 2026: Why Does Publish or Perish Apply to Patient Advocate Groups? The goal of this 1-hour CE-session was to identify why it is important to have industry, clinician and advocates involved; the importance of peer-reviewed published data; describe several […]
WORLDSymposium 2026 had 466 scientific abstracts presented at three (3) separate poster sessions. View 2026 Poster List.
WORLDSymposium 2026 Roscoe O. Brady Award was presented to Peter Marks, MD, PhD on Tuesday, February 3 at 08:00 PST, followed by a scientific presentation by Dr. Marks: Accelerating the Global Pace of Progress for Rare Diseases. View details.
Bob Stevens Received the 2026 Patient Advocate Leader (PAL) Award After reviewing numerous nominations, and considering many amazing individuals, the WORLDSymposium 2026 Awards Committee selected Bob Stevens to receive of the 2026 Patient Advocate Leader (PAL) Award. Bob Stevens is a globally respected leader in the lysosomal disease community, currently serving as Group CEO of […]
2026 WORLDSymposium Young Investigator Awards Congratulations to the ten individuals selected that received the 2026 WORLDSymposium Young Investigator Award. The award was a partial scholarship towards attendance at WORLDSymposium. 87 investigators-in-training submitted an application for the award. The depth and high quality of submissions made the Committee’s decisions especially challenging, a reflection of the remarkable talent advancing […]
Professor Michael H. Gelb, PhD, Received the 2026 Catalyst Award WORLDSymposium was delighted to announce Professor Michael Gelb, PhD, as the recipient of the 3rd Annual Catalyst Award that was presented at WORLDSymposium 2026. A chemist by training, Dr. Gelb has spent the second half of his career researching the development of new assays for expanded Newborn Screening with […]
Dr. Michael H. Gelb’s Keynote Address: Biochemical Newborn Screening for All Treatable Lysosomal Diseases, was on Thursday, February 5, 2026, at 08:00 PST. Read speaker bio.
5th Annual Robert J. Gorlin Symposium Global Access to Newborn Screening: A Call to Action! Thursday, February 5, 202617:45 – 19:00 PST1.25 Hour CE Session* Overview: There is tremendous global variation in access to newborn screening (NBS), even at the state or province level within individual countries, like the United States and Canada. With the […]
The Patient Voice 2026 Why Does Publish or Perish Apply to Patient Advocate Groups? Course Director: Jeanine R. Jarnes, PharmD, MSc, BCOP, BCPSMonday, February 2, 202615:00 – 16:00 PST1.00 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 […]
WORLDSymposium 2026 Mentor List Registered attendees met with these remarkable Mentors on Monday, February 2, 2026. Heather Adams, PhD Dr. Adams (Professor, Department of Neurology, URMC). A pediatric neuropsychologist and child psychologist, she studies natural history/neurocognitive phenotyping, quality of life, and adaptive function of NCL disorders and other childhood neuronopathic rare diseases. She is a past […]
2026 Industry Expert Theaters Monday, February 2, 2026, 16:30 – 17:30Hunter Syndrome: Advancing Understanding and Diagnosis in Neuronopathic MPS IIDownload Invitational PDFSponsored by NS Pharma Tuesday, February 3, 2026, 15:45 – 16:45From Experience to Insight: Transforming Care with Lived ExperienceSponsored by Amicus TherapeuticsDownload Invitational PDF Wednesday, February 4, 2026, 15:45 – 16:45Fabry Disease Reframed: Advancing […]
2026 Satellite Symposia Schedule Tuesday, February 3, 2026, 06:45-07:45Transforming Ambiguity to Action for Personalized Fabry CareSponsored by Amicus TherapeuticsClick Here to Download Informational PDF Tuesday, February 3, 2026, 06:45-07:45The maturation of evidence: what time teaches us about MPS IISponsored by Takeda Pharmaceutical Company LimitedClick Here to Download Informational PDF Tuesday, February 3, 2026, 12:15-13:15Evolving Landscape […]
The 2026 Symposium featured 35 exhibiting companies and 13 Patient Advocate Groups. View 2026 Exhibitor List.