WORLDSymposium announces James M. Wilson, MD, PhD, President and CEO of GEMMA Biotherapeutics, to receive the 2025 Roscoe O. Brady Award on Tuesday, February 4, 2025 at 08:00 PST. worldsymposia.org/worldsymposium… #WORLDSymposia #RareDisease #CareAboutRare

Please Share: WORLDSymposium 2025 Late-breaking Call for Abstracts Now Open. #WORLDSymposia will be accepting late-breaking abstracts from Nov. 1 to Dec. 2, 2024 for poster presentation & possible platform presentation. More at worldsymposia.org/worldsymposium… #RareDisease #CareAboutRare

Congratulations to the ten individuals selected to receive the WORLDSymposium 2025 Young Investigator Award. Visit worldsymposia.org/worldsymposium… to see the list of award recipients. #WORLDSymposia #RareDiseases #CareAboutRare

Late-breaking Abstract Submission closing next Monday, Dec. 2 - Only 5 days left to submit late-breaking abstracts and last chance to have platform presentation at WORLDSymposium 2025. Visit worldsymposia.org/worldsymposium… to submit your abstracts today! #WORLDSymposia #LysosomalDisease

#WORLDSymposia Late-breaking abstract submission closes today, December 2, 2024 at 12:00 CST. This is the last opportunity to submit abstracts for poster and/or presentation at WORLDSymposium 2025. Submit abstracts today: worldsymposia.org/worldsymposium… #RareDiseases #LysosomalDisease

WORLDSymposium announces Ryan Colburn to receive the 2025 Catalyst Award. Ryan will receive the 2nd Annual Catalyst Award at the Be the Catalyst event at 18:00 PST on Monday, February 3, 2025. worldsymposia.org/worldsymposium… #WORLDSymposia #RareDisease #Lysosomal Disease

Early Registration for #WORLDSymposia 2025 closes Monday, January 6, 2025. This includes reduced rates for The Patient Voice 2025 and On Demand. Click here to register: cvent.me/Ll4oYm #CareAboutRare #RareDiseases #LysosomalDisease

#WORLDSymposia 2025 Poster List Now Online. Over 400 abstracts will be presented at three poster sessions. Excellent opportunity to see, hear & discuss specific research topics directly with authors. #LysosomalDisease #CareAboutRare #RareDiseases worldsymposia.org/?p=94

#WORLDSymposia celebrated a pivotal moment, presenting the 2nd Annual "Catalyst" award to Ryan Colburn in recognition of his efforts to instigate a shift in perspective for the prevalence of Pompe disease #RareDiseases #LysosomalDisease #CareAboutRare

Dr. James M. Wilson was awarded the 2025 Roscoe O. Brady Award at #WORLDSymposia on Feb 4. Recognized as a global leader in gene therapy research and development. Congratulations, Dr. Wilson! #RareDiseases #LysosomalDisease #CareAboutRare

Congratulations to Maria Kefalas, the recipient of the #WORLDSymposium 2025 Patient Advocate Leader Award! Maria's mission to get an approved therapy for MLD has been instrumental in transforming MLD research. #RareDiseases #LysosomalDisease

Congratulations to Dean & Teryn Suhr, the recipient of the #WORLDSymposia 2025 Patient Advocate Leader Award! The Suhrs' were recognized for their tireless efforts to advance metachromatic leukodystrophy research and awareness. #RareDiseases #LysosomalDisease

Congratulations to the 2025 #WORLDSymposia Young Investigator Award rcpts: Pratikshya Adhikari, Aimee Donald, Allan Feng, Angelica Maria Herreno Pachon,  Andres Felipe Leal, Connor Lewis, Anna Reinelt, Krystyna Noelle Rytel, Roselena Schuh & Ewa Ziolkowska worldsymposia.org/worldsymposium…

2025 #WORLDSymposia New Treatment Award was presented to IntraBio for the FDA approval of AQNEURSA™ (levacetylleucine) for the treatment of neurological manifestations of #NiemannPick disease type C (NPC) in adults and peds weighing ≥15 kg. tinyurl.com/2p8b5uuc #RareDiseases

2025 #WORLDSymposia New Treatment Award was presented to Orchard Therapeutics for receiving FDA Approval of LENMELDY™ (atidarsagene autotemcel) for the treatment of children with PSLI, PSEJ, or ESEJ (collectively early-onset #MLD) tinyurl.com/2p8b5uuc #RareDiseases

2025 #WORLDSymposia New Treatment Award was presented to Zevra for FDA approval of MIPLYFFA™ (arimoclomol) capsules for use in combination w/ miglustat for neuro symptoms of #NiemannPick type C (NPC) in pts ≥2 yrs. tinyurl.com/2p8b5uuc #RareDiseases

Planning is underway for the 22nd Annual WORLDSymposium™, taking place February 2–6, 2026 in San Diego. This global meeting will highlight advances in lysosomal disease diagnosis, therapy, and translational research. Key dates: hubs.li/Q03t1zpN0 #WORLDSymposia

Sponsorship and exhibitor opportunities for WORLDSymposium™ 2026 are now available. Explore the prospectus and planning details. hubs.li/Q03vqc060 #WORLDSymposia #MedicalResearch #RareDisease

WORLDSymposium 2026 award nominations are now open. Nominate by September 3 for the: • Roscoe O. Brady Award • Patient Advocate Leader Award • New Treatment Award • Catalyst Award Details and nomination form: hubs.li/Q03y4JsT0

Nominate by Sept 3 for WORLDSymposium 2026 awards: • Roscoe O. Brady • PAL • New Treatment • Catalyst Details: hubs.li/Q03CsDlv0 #WORLDSymposia #ScientificRecognition