2023 NEW TREATMENT AWARD ANNOUNCED 

WORLDSymposium is pleased to announce the recipients of the 2023 New Treatment Award. Congratulations! Read full announcement here.

2023 YOUNG INVESTIGATOR AWARDS ANNOUNCED 

WORLDSymposium is pleased to announce the recipients of the 2023 Young Investigator Award. Congratulations to this year’s recipients! Read full announcement here.

PETER MARKS, MD, PhD RETURNS AS KEYNOTE SPEAKER ON FRIDAY, FEBRUARY 24, 2023 

WORLDSymposium is excited to announce the return of Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration, as the 2023 Keynote Speaker. Dr. Marks gave presentations to the WORLDSymposium audience in 2020 and 2021, providing important updates on the FDA’s role in rare disease research, and he will present the 2023 Keynote Address on Friday, February 24, 2023.

Dr. Marks’ Keynote Address: Taking Gene Therapy to the Next Level, on Friday, February 24, 2023 at 7:30 AM EST, at the 19th Annual WORLDSymposium in Orlando, Florida. Read full announcement here.

WORLDSymposium ANNOUNCES CHRISTINE WAGGONER AS RECIPIENT OF 2023 PATIENT ADVOCATE LEADER AWARD 

Congratulations to Christine Waggoner, the recipient of the WORLDSymposium 2023 Patient Advocate Leader (PAL) Award. After reviewing numerous nominations, and considering many amazing individuals, the WORLDSymposium 2023 Awards Committee has selected Christine as the recipient of the 2023 PAL Award. Christine founded the Cure GM1 Foundation in April 2015 in honor of her daughter Iris and all those affected by GM1 gangliosidosis. Christine’s work at Cure GM1 has involved a broad range, including animal models, biomarkers, gene therapy, enzyme replacement therapy, patient registries, patient reported outcomes, newborn screening and the first-ever GM1 caregiver preferences study.  Read full announcement here.

WORLDSymposium ANNOUNCES WILLIAM A. GAHL, MD, PHD, AS RECIPIENT OF 2023 ROSCOE O. BRADY AWARD

Each year, WORLDSymposium recognizes one individual for innovation and accomplishment in the field of lysosomal disease research and therapy. After reviewing numerous nominations, and considering many amazing individuals, the WORLDSymposium 2023 Awards Committee has selected William A. Gahl, MD, PhD, as the recipient of the 2023 Roscoe O. Brady Award. Dr. Gahl is the Director of the Undiagnosed Diseases Program, a Senior Investigator in the Medical Genetics Branch and the Head of the Human Biochemical Genetics Section of the National Human Genome Research Institute (NHGRI). Read full announcement here.

Plan to attend the 19th Annual WORLDSymposium Research Meeting February 22 – 26, 2023 at the Hilton Orlando in Orlando, FL, USA.

This symposium is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances.

Plans are in place for a LIVE in-person gathering in Orlando at the Hilton Orlando Hotel. In addition, plans are underway to make the program available ON DEMAND after the conference ends to allow for registration for those who are unable to attend in person.

Preliminary Schedule at a Glance

Schedule is subject to change
Download: WORLDSymposium 2023 Schedule at a Glance