Amicus Therapeutics Announces Presentations and Posters at the 21st Annual WORLDSymposium™ 2025
Amicus Therapeutics (FOLD) announced its participation at the 21st Annual WORLDSymposium™ 2025, featuring two oral presentations and 20 posters focused on Fabry and Pompe diseases. The presentations include key findings on miglustat, a first-in-class enzyme stabilizer for late-onset Pompe disease, and improvements in patients switching to cipaglucosidase alfa plus miglustat treatment.
The extensive poster sessions cover various aspects of both diseases, including patient experiences, treatment outcomes, and clinical studies. Notable presentations include long-term safety data of migalastat in Fabry disease patients, real-world evidence studies, and results from the PROPEL study showing clinical improvements in Pompe disease patients.
Amicus Therapeutics (FOLD) ha annunciato la sua partecipazione al 21° WORLDSymposium™ 2025, con due presentazioni orali e 20 poster focalizzati sulle malattie di Fabry e Pompe. Le presentazioni includono risultati chiave su miglustat, un stabilizzatore enzimatico di prima classe per la malattia di Pompe a esordio tardivo, e miglioramenti nei pazienti che passano al trattamento con cipaglucosidasi alfa più miglustat.
Le ampie sessioni di poster coprono vari aspetti di entrambe le malattie, incluse le esperienze dei pazienti, i risultati dei trattamenti e gli studi clinici. Tra le presentazioni più significative vi sono i dati di sicurezza a lungo termine di migalastat nei pazienti affetti da malattia di Fabry, studi su evidenze reali e risultati dello studio PROPEL che mostrano miglioramenti clinici nei pazienti con malattia di Pompe.
Amicus Therapeutics (FOLD) anunció su participación en el 21º WORLDSymposium™ 2025, que contará con dos presentaciones orales y 20 carteles centrados en las enfermedades de Fabry y Pompe. Las presentaciones incluirán hallazgos clave sobre miglustat, un estabilizador enzimático de primera clase para la enfermedad de Pompe de inicio tardío, y mejoras en pacientes que cambian a un tratamiento con cipaglucosidasa alfa más miglustat.
Las extensas sesiones de carteles cubrirán varios aspectos de ambas enfermedades, incluidas las experiencias de los pacientes, los resultados de los tratamientos y los estudios clínicos. Presentaciones notables incluyen datos de seguridad a largo plazo de migalastat en pacientes con enfermedad de Fabry, estudios de evidencia real y resultados del estudio PROPEL que muestran mejoras clínicas en pacientes con enfermedad de Pompe.
아미쿠스 테라퓨틱스(Amicus Therapeutics, FOLD)는 2025년 제21회 WORLDSymposium™에 참가한다고 발표했으며, 여기에는 파브리병 및 폼페병에 관한 두 개의 구두 발표와 20개의 포스터가 포함됩니다. 발표에는 미글루스타트(miglustat)에 대한 주요 발견이 포함되며, 이는 늦은 발병형 폼페병 치료를 위한 최초의 효소 안정제이며, 미글루스타트 치료로 전환한 환자들에서의 개선 사항도 포함됩니다.
광범위한 포스터 세션에서는 두 질병의 다양한 측면, 환자의 경험, 치료 결과 및 임상 연구가 다루어집니다. 주목할 만한 발표로는 파브리병 환자에서 미갈라스타트(migalastat)의 장기 안전성 데이터, 실제 증거 연구 및 폼페병 환자의 임상 개선 결과를 보여주는 PROPEL 연구 결과가 있습니다.
Amicus Therapeutics (FOLD) a annoncé sa participation au 21ème WORLDSymposium™ 2025, qui comprendra deux présentations orales et 20 affiches axées sur les maladies de Fabry et de Pompe. Les présentations incluent des résultats clés sur miglustat, un stabilisateur enzymatique de première classe pour la maladie de Pompe à début tardif, ainsi que des améliorations chez les patients passant à un traitement par cipaglucosidase alfa plus miglustat.
Les vastes sessions d'affiches couvrent divers aspects des deux maladies, y compris les expériences des patients, les résultats des traitements et les études cliniques. Les présentations notables incluent des données de sécurité à long terme de migalastat chez les patients atteints de la maladie de Fabry, des études d'évidence dans le monde réel et des résultats de l'étude PROPEL montrant des améliorations cliniques chez les patients atteints de la maladie de Pompe.
Amicus Therapeutics (FOLD) gab die Teilnahme am 21. WORLDSymposium™ 2025 bekannt, bei dem zwei mündliche Präsentationen und 20 Poster zu den Krankheiten Fabry und Pompe vorgestellt werden. Zu den Präsentationen gehören wichtige Ergebnisse zu miglustat, einem ersten enzymatischen Stabilator für die spät einsetzende Pompe-Krankheit, und Verbesserungen bei Patienten, die zu einer Behandlung mit Cipaglucosidase Alfa plus Miglustat gewechselt haben.
Die umfangreichen Postersitzungen decken verschiedene Aspekte beider Krankheiten ab, einschließlich der Patientenerfahrungen, Behandlungsergebnisse und klinischer Studien. Zu den bemerkenswerten Präsentationen gehören Langzeitdaten zur Sicherheit von Migalastat bei Patienten mit Fabry-Krankheit, Studien zur realen Evidenz und Ergebnisse der PROPEL-Studie, die klinische Verbesserungen bei Patienten mit Pompe-Erkrankung zeigen.
- Significant clinical improvements demonstrated in PROPEL study for Pompe disease patients
- Long-term safety and efficacy data available for migalastat in adolescent Fabry patients
- Comprehensive research portfolio with 22 total presentations showing strong R&D pipeline
- None.
PRINCETON, N.J., Feb. 03, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that two oral presentations and 20 posters across its development programs will be included at the 21st Annual WORLDSymposium™ 2025, being held February 3-7, 2025 in San Diego, CA.
“As part of our ongoing efforts to advance our scientific leadership in Fabry and Pompe diseases, Amicus researchers will present two oral presentations and 20 posters at this year’s WORLDSymposium,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc. “We’re proud of the progress we are making for patients living with Fabry and Pompe and are committed to build on this momentum in pursuit of our mission to develop and deliver transformative medicines for people living with rare diseases.”
Oral Platform Presentation:
Pompe Disease:
Abstract Title: Miglustat: A first-in-class enzyme stabilizer for late-onset Pompe disease
- Presenter: Robert J. Hopkin, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, U.S.A.
- Date and time: Wednesday, February 5, 11:00 a.m. PST
Abstract Title: Clinically important improvements in 6-minute walk distance (6MWD) and forced vital capacity (FVC) in adults with late-onset Pompe disease (LOPD) switching from alglucosidase alfa (alg) to cipaglucosidase alfa plus miglustat (cipa+mig) in the PROPEL study
- Presenter: Benedikt Schoser, Ludwig-Maximilians-University, Munich, Germany
- Date and time: Thursday, February 6, 1:30 p.m. PST
Poster Sessions:
Fabry Disease:
Abstract Title: Exploring the lived experiences and care perceptions of females living with Fabry in the US, Canada, and Japan (Poster #8)
- Presenter: Julia Alton, Canadian Fabry Association, Ontario, Canada
- Date and time: Tuesday, February 4, 3:30 – 5:30 p.m. PST
- Location: Kiosk 2-B
Abstract Title: Exploring the demographics of the diagnosed Fabry population in Canada (Poster #9)
- Presenter: Julia Alton, Canadian Fabry Association, Ontario, Canada
- Date and time: Tuesday, February 4, 3:30 – 5:30 p.m. PST
- Location: Kisok 3-A
Abstract Title: Stable: A protein engineering platform for enhancing stability and delivery of alpha-galactosidase A for Fabry disease therapy (Poster #358)
- Presenter: Tobias Willer, Amicus Therapeutics, Philadelphia, PA, U.S.A.
- Date and time: Wednesday, February 5, 3:30 – 5:30 p.m. PST
- Location: Kiosk 31-A
Abstract Title: Long-term safety and efficacy of migalastat in adolescent patients with Fabry disease: Results from the ASPIRE study and open-label extension (Poster #283)
- Presenter: Uma Ramaswami, Royal Free London NHS Foundation Trust and Genetics and Genomic Medicine, London, U.K.
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kiosk 32-D
Abstract Title: Treatment satisfaction in patients with Fabry disease: Patient-reported outcomes from the FollowME Fabry Pathfinders registry and the SATIS-Fab study (Poster #90)
- Presenter: Ulla Feldt-Rasmussen, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kiosk 1-C
Abstract Title: FollowME Fabry Pathfinders Registry: Patient-reported outcomes in a cohort of patients on migalastat treatment for median 4 years (Poster #91)
- Presenter: Ulla Feldt-Rasmussen, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kiosk 1-D
Abstract Title: Quality of life of migalastat-treated adolescents with Fabry disease: Results from the ASPIRE study and open-label extension (Poster #142)
- Presenter: Robert J. Hopkin, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, U.S.A.
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: 15-C
Abstract Title: Trial in progress: An open-label study (AT1001-025 RENEW) to evaluate the safety and pharmacokinetics of migalastat in patients with Fabry disease and amenable GLA variants with severe renal impairment or end-stage renal disease treated with dialysis (Poster #136)
- Presenter: Jennifer Hiros, Amicus Therapeutics, Princeton, NJ, U.S.A.
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kisok 12-D
Abstract Title: An observational cohort study evaluating outcomes in patients with Fabry disease treated with migalastat in a real-world setting in France (MIGA-Fab) (Poster #185)
- Presenter: Didier Lacombe, Bordeaux University Hospital, Bordeaux, France
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kiosk 24-C
Pompe Disease:
Abstract Title: Non-invasive magnetic resonance imaging monitoring of glycogen accumulation in a mouse model of Pompe disease (Poster #361)
- Presenter: Nirbhay Yadav, F. M. Kirby Research Center for Functional Brain Imaging, Kennedy Krieger Institute, Baltimore, MD, U.S.A.
- Date and time: Tuesday, February 4, 3:30 – 5:30 p.m. PST
- Location: Kiosk 21-D
Abstract Title: Miglustat: A first-in-class enzyme stabilizer for late-onset Pompe disease (Poster #139)
- Presenter: Robert J. Hopkin, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, U.S.A.
- Date and time: Wednesday, February 5, 3:30 – 5:30 p.m. PST
- Location: Kiosk 12-A
Abstract Title: Reaching consensus on comprehensive and achievable monitoring for adults with late-onset Pompe disease in the UK (Poster #71)
- Presenter: Patrick Deegan, Cambridge University Hospitals NHS Foundation Trust (CUH), Cambridge, U.K.
- Date and time: Wednesday, February 5, 3:30 – 5:30 p.m. PST
- Location: Kiosk 4-D
Abstract Title: Pompe disease in Sweden: A real-world evidence study investigating disease burden, treatment patterns for enzyme replacement therapy and concomitant medications (Poster #248)
- Presenter: Sofia Nordin, Amicus Therapeutics, Copenhagen, Denmark
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kiosk 26-D
Abstract Title: Patient perspective of participation in clinical studies in LOPD: Structured exit interviews from cipaglucosidase alfa plus miglustat studies (Poster #102)
- Presenter: Jamie Gault, Amicus Therapeutics, Princeton, NJ, U.S.A.
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kisok 15-B
Abstract Title: POM-005: A global, prospective, observational registry of people living with Pompe disease (Poster #225)
- Presenter: Paul McIntosh, University of Pennsylvania, Philadelphia, PA, U.S.A.
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kiosk 30-A
Abstract Title: Clinically important improvements in 6-minute walk distance (6MWD) and forced vital capacity (FVC) in adults with late-onset Pompe disease (LOPD) switching from alglucosidase alfa (alg) to cipaglucosidase alfa plus miglustat (cipa+mig) in the PROPEL study (Poster #303)
- Presenter: Benedikt Schoser, Ludwig-Maximilians-University, Munich, Germany
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kiosk 36-B
Abstract Title Outcomes of a pediatric patient with late-onset Pompe disease switching from high-dose, high-frequency alglucosidase alfa to standard-dose cipaglucosidase alfa plus miglustat (Poster #70)
- Presenter: Stephanie DeArmey, Duke University Medical Center, Durham, NC, U.S.A.
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kiosk 5-C
Abstract Title: Cipaglucosidase alfa plus miglustat in late-onset Pompe disease: two non-ambulatory patients switching from high-dose, high-frequency alglucosidase alfa (Poster #46)
- Presenter: Barry J. Byrne, University of Florida, Gainesville, FL, U.S.A.
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kiosk 7-B
Abstract Title: Post-baseline outcomes of the UK Early Access to Medicines Scheme registry for cipaglucosidase alfa plus miglustat in late-onset Pompe disease (Poster #288)
- Presenter: Mark Roberts, Salford Royal NHS Foundation Trust, Salford, U.K.
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kiosk 33-B
Abstract Title: Correction of glycogen accumulation in muscle, heart and CNS in a pre-clinical model of hematopoietic stem cell gene therapy for Pompe disease (Poster #351)
- Presenter: Slawomir Wantuch, UCL Great Ormond Street Institute of Child Health, London, U.K.
- Date and time: Thursday, February 6, 3:30 – 5:30 p.m. PST
- Location: Kisok 38-D
About WORLDSymposium
WORLDSymposium 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. Each year, WORLDSymposium presents the latest information from basic science, translational research, and clinical trials for lysosomal diseases. For more information, please visit www.worldsymposia.org.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on X and LinkedIn.
CONTACTS:
Investors:
Amicus Therapeutics
Andrew Faughnan
Vice President, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
Media:
Amicus Therapeutics
Diana Moore
Vice President, Corporate Communications
dmoore@amicusrx.com
(609) 662-5079
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