2023 NEW TREATMENT AWARD ANNOUNCED 

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

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2023 YOUNG INVESTIGATOR AWARDS ANNOUNCED 

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

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PETER MARKS, MD, PhD RETURNED 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 presented the 2023 Keynote Address on Friday, February 24, 2023.

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

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WORLDSymposium ANNOUNCED 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.

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WORLDSymposium ANNOUNCED 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.

Preliminary Schedule at a Glance

Day	Tuesday Feb. 21, 2023	Wednesday Feb. 22, 2023	Thursday Feb. 23, 2023	Friday Feb. 24, 2023	Saturday Feb. 25, 2023	Sunday Feb. 26, 2023
		Satellite Symposia	Satellite Symposia	Satellite Symposia	Satellite Symposia
Morning		Roscoe. O. Brady Award Basic Science	Patient Advocate Leader & Young Investigator Awards Translational Research	Keynote Address Clinical Applications	New Treatment Award Contemporary Forum (Non-CME)	Late-breaking Science
Midday		Satellite Symposia	Satellite Symposia	Satellite Symposia	Satellite Symposia	11:15 AM Adjourn
Afternoon	Robert J. Gorlin Symposium	Basic Science	Translational Research	Clinical Applications	Contemporary Forum (Non-CME)
	Emerging Trends	Poster Session I & II	Poster Session III & IV	Poster Session V & VI	Poster Session VII & VIII
Evening	Be the Catalyst Event	Speed Mentoring Event	Satellite Symposia	Satellite Symposia