WORLDSymposium™ Online 1-hour CME Session: Cardiovascular Implications in Fabry Disease Now Available

WORLDSymposium has released a free one-hour online CME session “Cardiovascular Implications in Fabry Disease: A Multidisciplinary Approach”, available to all health care practitioners. The course is available at CME University and at myCME.

There is a required pre-test and post-test in order to obtain the credit.

If you are already registered with CME University or myCME, you may use your user name and password to login and complete the activity. If you are a new user, you will be prompted to set up a login so you can save your progress and credit history.


WORLDSymposium™ 2020 will feature over 480 scientific abstracts presented at three separate poster sessions. These sessions are an excellent opportunity to see, hear and discuss specific research topics directly with the abstract authors. Click here for more information and to download the list.

Celebrating 15 years of Naglazyme® (galsulfase)

Please join BioMarin in celebrating 15 years of dedication to MPS and listen to the stories that detail the journey of how one of the treatments came to fruition. This evening reception will be in the Windermere Ballroom on Monday, February 10, from 6:30-8:00 PM and is open to all attendees.

Enjoy some light refreshments and hear directly from some of the clinicians, first clinical trial patients, as well as BioMarin as they share what this treatment has meant to the community.


WORLDSymposium is pleased to announce Chester B. Whitley, PhD, MD, will present the Keynote Address Navigating Clinical Trials on Wednesday, February 12, 2020 at 7:45 AM.

Dr. Whitley is Professor of Pediatrics and a Faculty Member of both the Division of Genetics and Metabolism, and the Department of Experimental and Clinical Pharmacology at the University of Minnesota Medical School in Minneapolis, Minnesota. He is certified by the American Board of Pediatrics and the American Board of Medical Genetics and Genomics in the areas of Clinical Biochemical Genetics and Clinical Genetics.

Click here for more information about the Keynote Speakers.


The Preliminary Program for WORLDSymposium 2020 is now available. Four full days of new discoveries, cutting-edge research, and updates from ongoing studies will be presented by scientists and clinicians from around the globe, including 12 ground-breaking presentations from industry leaders in the New Contemporary Forum.

WORLDSymposium Young Investigator Awards Announced

Congratulations to the eleven individuals selected to receive the WORLDSymposium Young Investigator Award for 2020. The award is a partial scholarship towards attendance at WORLDSymposium 2020. WORLDSymposium would like to congratulate all of the applicants for their hard work. Award recipients will be recognized at the Opening Session of the Scientific Meeting on Monday, February 10, 2020 at 1:00 PM in the General Session. Click here for the full announcement.

Call for Abstracts: WORLDSymposium 2020 Online Late Breaking Abstract Submission Now Closed

Late-breaking abstract submissions for WORLDSymposium 2020 are now closed.

WORLDSymposium was accepting late-breaking abstracts from November 1 to December 1, 2019 for poster presentation only. (Late-breaking abstracts are published in the onsite program materials, and are not published in Molecular Genetics and Metabolism.)

Click here for more information about late-breaking abstract submissions.

Peter Marks to Deliver Keynote Address on Thursday, February 13, 2020

Peter Marks WORLDSymposium 2020 Keynote SpeakerThe WORLDSymposium Planning Committee is excited to announce Peter Marks, MD, PhD, as Keynote Speaker on Thursday, February 13, 2020 at the 16th Annual WORLDSymposium Scientific Meeting in Orlando, Florida, USA.

Dr. Marks is the director of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA). The center is responsible for assuring the safety and effectiveness of biological products, including vaccines, allergenic products, blood and blood products, and cellular, tissue, and gene therapies.

Dr. Marks will be presenting “The Shift from Personalized to Individualized Therapies” as the Keynote Address on Thursday, February 13, 2020 at 7:30 AM. Plan to arrive early on Thursday, as seating is expected to fill quickly for his important messages.

Click here to read more.

WORLDSymposium Announces John F. Crowley as Recipient of 2020 Roscoe O. Brady Award for Innovation and Accomplishment

John F. Crowley, Amicus TherapeuticsEach year, WORLDSymposium requests nominations from the lysosomal disease community, and then 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 2020 Planning Committee selected nominee John F. Crowley to receive the 2020 Roscoe O. Brady Award for Innovation and Accomplishment.

The 2020 Roscoe O. Brady Award for Innovation and Accomplishment will be presented to John F. Crowley, Chairman of the Board, Chief Executive Officer, Amicus Therapeutics, Inc. on Tuesday, February 11, 2020 at 7:30 AM.

Click here to read more.

WORLDSymposium Announces Cara O’Neill, MD as Recipient of the 2020 Patient Advocate Leader (PAL) Award

Cara O’Neill, MD - Patient Advocate Leader Award Recipient 2020Each year, WORLDSymposium recognizes one individual for patient advocacy leadership in the field of lysosomal disease. The 2020 Patient Advocate Leader (PAL) award will be presented to Cara O’Neill, MD, founder and Chief Science Officer at Cure Sanfilippo Foundation on Wednesday, February 12, 2020 at 7:30 AM.

Dr. ONeill and her husband founded Cure Sanfilippo Foundation after receiving her daughter’s diagnosis in 2013, guided by the mission to support the creation of treatments and an eventual cure for the disease. Since then, they have spread awareness around the globe about Sanfilippo syndrome via talk shows, news media, online platforms, and international newspapers.

Click here to read more.

Monday, February 10, 2020: Opening Reception and Poster Session I

This year, Monday evening will include an Opening Reception and the first of three Poster Sessions in the Exhibit Hall. Plan to arrive early for this special event.

Claiming Educational Credits for the 2019 WORLDSymposium Meeting

Information on how to claim education credits is given only to registered attendees and is available in the 2019 WORLDSymposium mobile app or by contacting us.

Preliminary Schedule at A Glance

Day Monday
Feb. 10, 2020
Feb. 11, 2020
Feb. 12, 2020
Feb. 13, 2020
6:15-7:15 AM Satellite Symposia Satellite Symposia Satellite Symposia
Morning Emerging Trends 2020 (8:00-11:30 AM) Basic Science: Disease Mechanisms, Pathology, and Biomarkers Translational Research Clinical Trial Outcomes2
Lunch Satellite Symposia Satellite Symposia Satellite Symposia Satellite Symposia
Afternoon Basic Science: Disease Mechanisms, Pathology, and Biomarkers Translational Research Clinical Trials for Registration1 NEW Contemporary Forum: Industry Abstracts from All Categories (Non-CME)
4:30-6:30 PM Opening Reception & Poster Reception I Poster Reception II Poster Reception III Networking Reception (4:30-5:30 PM)
6:30-8:30 PM Satellite Symposia Satellite Symposia Satellite Symposia Lysosomal Disease Network (LDN) COPA and Council of Research Experts (CORE) (5:00 – 7:00 PM)
1 Abstracts in the Clinical Trials for Registration category must have an FDA Investigational New Drug (IND) application (Phase I-III clinical trial), an EMA Investigational Medicinal Product Dossier (IMPD), or equivalent.
2 The Clinical Outcomes category includes licensed therapies (Phase IV) and non-interventional studies.

We’re Organizing Research on Lysosomal Diseases

Dates to Remember

July 25, 2019
Abstract submission website open.

August 1, 2019
Online registration and hotel reservations open.

August 9, 2019
Exhibitor prospectus available by request.

September 1, 2019
Deadline for submission of full-length manuscripts for peer-review in the “Lysosomes” issue of Molecular Genetics & Metabolism, February, 2020. Click here to access the Guide for Authors or to Submit Your Paper.

October 1, 2019
Deadline for abstract submission to WORLDSymposium 2020.

November 1 – December 1,  2019
Late breaking abstract submission open.

November 5, 2019
Notification of abstract acceptance for poster or platform presentation at WORLDSymposium 2020.

About WORLDSymposium

The goal of WORLDSymposium is to provide an interdisciplinary forum to explore and discuss specific areas of interest, research and clinical applicability related to lysosomal diseases. Each year, WORLDSymposium hosts a scientific meeting presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases. This symposium is designed to help researchers and clinicians to better manage and understand diagnostic options for patients with lysosomal diseases, identify areas requiring additional basic and clinical research, public policy and regulatory attention, and identify the latest findings in the natural history of lysosomal diseases.

Sign up for Updates

Sign up for Updates

Click here to sign up for updates about the 16th Annual WORLDSymposium.

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.