WORLDSymposium 2024 Preliminary Program* on Lysosomal Diseases

The preliminary program for WORLDSymposium will be posted by the end of November, 2023.

WORLDSymposium has become the major educational and unifying activity of Lysosomal Disease Researchers, and has evolved into a highly interactive research activity. The underlying theme “transitioning molecular biology to human therapies” seeks to elucidate the challenges—and highlight the successes—in bringing bench discoveries into successful clinical therapies.

The main emphasis of the meeting remains the same: to assess the mechanisms and obstacles for taking bench research into human therapy.

The WORLDSymposium research program is focused on the 3 main areas of lysosomal disease research: basic and bench science, translational research, and clinical trials and outcomes. This progression through the three stages of research is a signature feature of WORLDSymposium and is enthusiastically received based on attendee evaluation.

Basic and Bench Science. After the presentation of the Roscoe O. Brady Award, and featured award recipient presentation, the Basic and Bench Science session will discuss innovations in technology and how they can be applied to early diagnosis for lysosomal conditions, progress in gene therapy, and exploitation of differences at the cellular level that may indicate early disease state.

Translational Research – Including Gene Therapy and Newborn Screening. Following the Patient Advocate Leader Award and the Young Investigator Awards, Translational Research sessions turn to the challenge of moving laboratory discoveries to therapy, the important hurdles of translational research. Some broad topics of discussion include modulation of CNS affects of disease, how to increase the efficacy of therapeutic modalities, and genotype/phenotype correlations.

Clinical Trials for Registration and Clinical Outcomes. These sessions are committed to presentations of results from clinical trials, in most cases, the actual application of new agents in humans affected by these conditions. These sessions will include presentations related to re-thinking the definition of biomarkers for lysosomal disease.

Industry Contemporary Forum (Non-CE). For abstract submissions that include first authors with a corporate affiliation (employment/salary or other unresolvable conflict), we will offer a day for platform presentations as a non-CE session to provide attendees with cutting-edge industry research, data and development. Information regarding the first author’s corporate affiliation has been added to the abstract submission site, and these abstracts will be reviewed and selected as part of the Contemporary Forum day.

*This will be a preliminary program only. ALL times and speakers will be subject to change. Be sure to check back weekly as updates are made.

Attention to All Platform Speakers: It is the policy of WORLDSymposium to publish all abstracts with the list of authors exactly as the abstract was submitted to WORLDSymposium. The first author of the submitted abstract will be listed as the Platform Speaker (presenting author) on the Preliminary Program, Agenda, and Poster List.

All requests for changes to the Platform Speaker (presenting author) will be reviewed by the WORLDSymposium Planning Committee prior to approval. No changes will be made to the Preliminary Program posted online; any changes to the Platform Speaker will be announced from the podium by the Moderators only at the time of the platform presentation. The Preliminary Program will remain published with the original first author, and all communication will continue to go to the first author of the abstract.

The submitting first author will continue to receive all instructions from the automated abstract notification system. Please be sure that the first author is aware they will need to forward any email instructions for PowerPoint preparation, and for advance review of presentations. Meeting the presentation guidelines and submitting presentations for prior review must be done to meet ACCME accreditation requirements.

Reference the Frequently Asked Questions pages for additional information.