WORLDSymposium 2024 New Treatment Award

2023 New Treatment Award WORLDSymposium
Chester B. Whitley, Bill Sibold, Monica Kumar, Alaa Hamed, Shannon Resetich, Paul Hudson

WORLDSymposium strives to recognize important achievements in therapy for lysosomal diseases for treatments attaining regulatory approval. In recognition of achieving major milestones, WORLDSymposium reviews potential recipients for the New Treatment Award in 2023. This award honors “new treatments” that are viewed as providing value to patients with lysosomal diseases, and general acceptance as evidenced by approval by the U.S. Food and Drug Administration, European Medicines Agency, and other regulatory authorities.

The WORLDSymposium New Treatment Award for 2023 was presented to Sanofi for olipudase alfa-rpcp (Xenpozyme™) which provided clinical data meriting approval by the U.S. Food and Drug Administration (FDA) as the first disease-specific treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. The New Treatment Award was presented on Saturday, February 25, 2023, at 7:45 AM EST, followed by a full day of Contemporary Forum abstract presentations.

William A. Gahl, MD, PhD Received the 2023 Roscoe O. Brady Award

View Dr. Gahl’s scientific presentation Pursuing Advances in Rare and Undiagnosed Diseases on YouTube.

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).

Dr. Gahl’s research has focused on the natural history of rare metabolic disorders and the discovery of new genetic diseases. He elucidated the basic defects in cystinosis and Salla disease, i.e., deficiencies of the lysosomal membrane transporters that carry cystine and sialic acid, respectively, out of the lysosome.

Dr. William A. Gahl graduated from the Massachusetts Institute of Technology and earned his M.D. and Ph.D. from the University of Wisconsin. He served as pediatric resident and chief resident at the University of Wisconsin hospitals and completed clinical genetics and clinical biochemical genetics fellowships at the NIH. Dr. Gahl elucidated the basic defects in cystinosis and Salla disease and helped bring cysteamine to new drug approval by the Food and Drug Administration as the treatment for cystinosis.

He has published over 650 papers, reviews, book chapters, and editorials, trained 42 biochemical geneticists and cultivated international experts in Hermansky-Pudlak syndrome, alkaptonuria, Oculocerebrorenal Syndrome of Lowe, Menkes disease, Congenital Disorders of Glycosylation, Griscelli Syndrome, Gray Platelet Syndrome, Joubert Syndrome, polycystic kidney disease and other ciliopathies, Hutchinson-Gilford Progeria, GNE myopathy, oculocutaneous albinism, sialuria, and free sialic acid storage disorders. His group identified the genes responsible for Hartnup disease, Gray Platelet Syndrome, two types of renal Fanconi syndrome, 3-methylglutaconic aciduria type III, a new neutrophil defect, and many other disorders.

In 2008, he established the NIH Undiagnosed Diseases Program (UDP), which has made more than 350 rare disease diagnoses and discovered 30 new genetic diseases. Dr. Gahl expanded the UDP to a national Undiagnosed Diseases Network and a worldwide Undiagnosed Diseases Network International. He established American Board of Medical Specialties certification for medical biochemical genetics. Dr. Gahl received the Dr. Nathan Davis Award for Outstanding Government Service from the AMA, the Service to America Medal in Science and the Environment, the EURORDIS Lifetime Achievement Award, and numerous other awards. In 2019, he was elected to the National Academy of Medicine.

Congratulations to Dr. Gahl from the entire WORLDSymposium audience! Dr. Gahl received the 2023 Roscoe O. Brady Award on Wednesday, February 22, 2023 at 7:30 AM EST, and this was followed by Dr. Gahl’s scientific presentation Pursuing Advances in Rare and Undiagnosed Diseases (view on YouTube).

WORLDSymposium registration is restricted to persons age 18 years and over only. This includes all Scientific Sessions and the Exhibit Hall.

The Registration Desk, located in the Palm Foyer on the Second Level of the Manchester Grand Hyatt San Diego, will be open during these times:

Sunday, February 4, 12:00 PM - 6:00 PM
Monday, February 5, 6:00 AM - 5:15 PM
Tuesday, February 6, 6:00 AM - 5:15 PM
Wednesday, February 7, 6:00 AM - 5:15 PM
Thursday, February 8, 6:00 AM - 5:15 PM
Friday, February 9, 7:30 AM - 10:00 AM

Appropriate dress is business casual. Please dress appropriately whenever attending in person session or appearing on pre-recorded or live video for presentations or when in video meetings.

Registration and attendance at, or participation in, WORLDSymposium Scientific Sessions and other related activities, constitutes an agreement by the registrant to permit WORLDSymposium use and distribution (both now and in the future) of the registrant or attendee’s image or voice in photographs, videotapes, electronic reproductions, audiotapes of such events and activities.

Attendees may take photos or screen captures of posters ONLY if the poster author agrees. Authors who do not want their posters to be photographed or screen captured will have to indicate as such on their posters. No other photography, or audio or video recording is allowed. Attendees who photograph or record poster information for which they have not obtained permission will be asked to leave the session immediately.

Taking photos, videos or audiotapes of slides, posters and presenters or questions and answers is strictly prohibited unless prior written permission is obtained from the presenter. Copyright and intellectual property law is observed during all WORLDSymposium presentations, sessions and events.

Attendees and media registrants are required to abide by any recording, photography, or screen capture requests made by speakers at any WORLDSymposium event. Photography and recording for personal reference is allowed. However, attendees and media registrants may not share or republish any materials from WORLDSymposium events, including but not limited to slides, unpublished data, or presentation materials, without explicit written permission from the presenter and WORLDSymposium. 

Poster Photo Policy: Attendees may take photos of posters ONLY if the poster author agrees. Authors who do not want their posters to be photographed will have to indicate as such on their posters. No other photography, or audio or video recording is allowed. Attendees who photograph or record poster information for which they have not obtained permission will be asked to leave the session immediately.

This policy aims to encourage presentation of the latest findings by protecting researchers sharing unpublished information, as well as prevent disruption in the session rooms.

Attendees are also asked to be respectful of their colleagues by silencing all mobile devices before entering meeting rooms. When registering, you are required to agree that you will adhere to this policy.

No.  There is no additional fee to attend the The Patient Voice: Is Anyone Listening? session, or the Robert J. Gorlin Symposium: Beyond the Blood Brain Barrier.

WORLDSymposium does not provide an exhibit hall-only registration fee. In order to visit the exhibits, an attendee must purchase a full conference registration. If you are interested in a great opportunity to connect with over 2000 key members of the international lysosomal disease community, WORLDSymposium is the place!

Hotel reservations for individuals who are able to attend the live conference in San Diego opens August 1, 2023. Click here for more information.

A limited block of discounted rooms ($279 + 12% tax and $37 fees) at the Manchester Grand Hyatt San Diego will be available until January 5, 2024 or when the room block fills, whichever comes first.

WORLDSymposium provides the following refreshments and meals, open to all registered attendees:

  • Light breakfast in the foyer (Monday-Friday)
  • Morning and afternoon refreshment breaks in the exhibit hall (Monday-Thursday)
  • Morning refreshment break in the foyer (Friday)
  • Boxed lunch in the foyer (Monday-Thursday)
  • Light refreshments and a cash bar in the foyer during evening networking events (Sunday and Monday)
  • Light refreshments and a cash bar in the exhibit hall during afternoon poster receptions (Monday-Thursday)
  • Light refreshments in the foyer during late afternoon satellites (Tuesday and Wednesday)

The On Demand component of WORLDSymposium has no meals included in the registration fee.

CANCELLATION POLICY: Cancellation notices must be in writing and received by 4:30 PM Central Standard Time on January 5, 2024 to be eligible for a refund. All cancellations are subject to a nonrefundable processing fee of 20% of all registration fees paid. No refunds will be made after this date. Cancellation notices should be sent to registration@nullworldsymposia.org. Allow four weeks for processing.

If a registrant needs to cancel after January 5, 2024, the conference registration may be transferred to another person who is NOT already registered for the meeting. If you have any questions, please contact registration@nullworldsymposia.org.

You will be able to change or update your registration by entering your email and your confirmation number.

  • Any time prior to January 5, 2024, any individual registered for the LIVE conference, may transfer their registration to the On Demand-only registration, and receive a full refund of the difference between the LIVE and On Demand rates. After January 5, 2024, any individual may still change their registration from LIVE to On Demand, but no refund will be applied. No exceptions.
  • Anyone who is registered for On Demand only who wants to upgrade to LIVE registration may also do so any time prior to January 5, 2024 and pay the difference between the two early registration rates. After January 5, 2024, the attendee will be required to pay the difference between the late registration fee and the original amount paid.
  • PLEASE NOTE: These changes can be done using the Modify Registration link. Please contact registration@nullworldsymposia.org if you have any issues changing registration from LIVE to On Demand or from On Demand to LIVE registration.

Registration payments can be made by check, credit card (American Express, MasterCard, Discover, or Visa only) or wire transfer. We do not process phone or fax registrations.

WORLDSymposium is intended for healthcare professionals involved in the screening, diagnosis, management, and treatment of lysosomal diseases affecting both children and adults, with research and clinically relevant information geared specifically to the following professionals:

  • Medical and clinical geneticists; genetic counselors; pediatricians; neurologists; psychologists; nurse practitioners; physician assistants; registered nurses; and all medical practitioners who are providing comprehensive diagnostic, management, and counseling services for patients with, or at risk for, lysosomal diseases.
  • Laboratory directors and technicians involved in genetic testing.
  • Researchers involved in lysosomal disorders and treatments.
  • Clinical, laboratory and research trainees of genetics and all biomedical sciences with a focus on lysosomal diseases.
  • Any healthcare and public health professionals who have an interest in medical and clinical genetics and genomics in the area of lysosomal diseases.
  • Advocates for patients with lysosomal diseases and their families.

NOTE: Adult patients and adult family members are welcome to attend the educational sessions, however the content is intended for clinicians and researchers, and not targeted toward patients. Attendance for patients and family members is offered as an opportunity to obtain information pertinent to their personal journey and to connect with leading researchers and clinicians. Children under 18 are not allowed in any session.

WORLDSymposium will be conducting the Annual Research Conference as a live event and will be held, in its entirety, at the Manchester Grand Hyatt San Diego in San Diego, California, USA.

Registration Opens: August 1, 2023.
Advance Registration Deadline: January 5, 2024.

It is mutually agreed that in the event WORLDSymposium is cancelled in whole or in part, or the scheduled opening is cancelled or delayed, due to causes that are beyond the reasonable control of WORLDSymposium, such as disasters, strikes, earthquakes, flood, civil disturbances, acts of God, acts of terrorism, health warning, advisory or directive, epidemic, pandemic, governmental regulations or any other circumstance beyond the control of WORLDSymposium that make it impossible or impractical to open or continue the event, then and thereupon the contract between Company and WORLDSymposium will automatically be terminated and Company hereby waives any and all claims against WORLDSymposium for damages, reimbursement, refunds or compensation.