Amicus Therapeutics Announces Participation at the SSIEM 2024 Annual Symposium
Amicus Therapeutics (Nasdaq: FOLD) announced its participation in the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium in Porto, Portugal, from September 3-6, 2024. The company will present five posters across its development programs, focusing on Fabry Disease and Pompe Disease.
The presentations include three posters on Fabry Disease, discussing patient-reported outcomes from the FollowME Fabry Pathfinders registry, quality of life in migalastat-treated adolescents, and the journey of patients living with Fabry disease in Poland. Two posters on Pompe Disease will cover post-baseline outcomes of the UK Early Access to Medicines Scheme registry for cipaglucosidase alfa plus miglustat, and miglustat as a first-in-class enzyme stabilizer for late-onset Pompe disease.
Amicus Therapeutics (Nasdaq: FOLD) ha annunciato la sua partecipazione al Simposio Annuale della Società per lo Studio degli Errori Congeniti del Metabolismo (SSIEM) 2024 che si terrà a Porto, Portogallo, dal 3 al 6 settembre 2024. L'azienda presenterà cinque poster relativi ai suoi programmi di sviluppo, concentrandosi su malattia di Fabry e malattia di Pompe.
Le presentazioni includeranno tre poster sulla malattia di Fabry, discutendo i risultati riportati dai pazienti del registro FollowME Fabry Pathfinders, la qualità della vita negli adolescenti trattati con migalastat e il percorso dei pazienti che vivono con la malattia di Fabry in Polonia. Due poster sulla malattia di Pompe tratteranno i risultati post-baseline del registro del Regno Unito per l’Accesso Anticipato ai Farmaci riguardante cipaglucosidase alfa con miglustat e miglustat come stabilizzatore enzimatico di prima classe per la malattia di Pompe a esordio tardivo.
Amicus Therapeutics (Nasdaq: FOLD) anunció su participación en el Símbolo Anual de la Sociedad para el Estudio de Errores Congénitos del Metabolismo (SSIEM) 2024 que se llevará a cabo en Oporto, Portugal, del 3 al 6 de septiembre de 2024. La compañía presentará cinco pósters de sus programas de desarrollo, con un enfoque en la enfermedad de Fabry y la enfermedad de Pompe.
Las presentaciones incluirán tres pósters sobre la enfermedad de Fabry, discutiendo los resultados reportados por los pacientes del registro FollowME Fabry Pathfinders, la calidad de vida en adolescentes tratados con migalastat, y el recorrido de los pacientes que viven con la enfermedad de Fabry en Polonia. Dos pósters sobre la enfermedad de Pompe cubrirán los resultados post-baseline del registro del Reino Unido para el Acceso Anticipado a Medicamentos con cipaglucosidasa alfa más miglustat, y miglustat como estabilizador enzimático de primera clase para la enfermedad de Pompe de inicio tardío.
아미쿠스 테라퓨틱스 (Nasdaq: FOLD)는 포르투갈 포르투에서 2024년 9월 3일부터 6일까지 열리는 선천성 대사 오류 연구학회의 연례 심포지움 (SSIEM) 2024에 참여한다고 발표했습니다. 이 회사는 파브리병 및 폼페병에 초점을 맞춘 다섯 개의 포스터를 발표할 예정입니다.
발표에는 파브리병에 대한 세 개의 포스터가 포함되어 있으며, 이는 FollowME Fabry Pathfinders 등록부에서 환자가 보고한 결과, 미갈라스타트로 치료받은 청소년의 삶의 질, 그리고 폴란드에서 파브리병을 앓고 있는 환자의 여정을 다룹니다. 폼페병에 대한 두 개의 포스터는 cipaglucosidase alfa와 miglustat에 대한 영국 조기 약물 접근 등록부의 기준선 이후 결과와, 후기 발현 폼페병에 대한 첫 번째 클래스 효소 안정제인 miglustat에 대해 다룰 것입니다.
Amicus Therapeutics (Nasdaq: FOLD) a annoncé sa participation au Symposium Annuel de la Société pour l'Étude des Erreurs Congénitales du Métabolisme (SSIEM) 2024 qui se tiendra à Porto, au Portugal, du 3 au 6 septembre 2024. L'entreprise présentera cinq affiches liées à ses programmes de développement, en mettant l'accent sur la maladie de Fabry et la maladie de Pompe.
Les présentations comprendront trois affiches sur la maladie de Fabry, discutant des résultats rapportés par les patients du registre FollowME Fabry Pathfinders, de la qualité de vie des adolescents traités par migalastat, et du parcours des patients vivant avec la maladie de Fabry en Pologne. Deux affiches sur la maladie de Pompe traiteront des résultats post-baseline du registre du Royaume-Uni pour l'Accès Précoce aux Médicaments concernant la cipaglucosidase alfa associée à miglustat, et miglustat en tant que stabilisateur enzymatique de première classe pour la maladie de Pompe à début tardif.
Amicus Therapeutics (Nasdaq: FOLD) gab seine Teilnahme am Jahressymposium der Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 in Porto, Portugal, vom 3. bis 6. September 2024 bekannt. Das Unternehmen wird fünf Poster zu seinen Entwicklungsprogrammen präsentieren, wobei der Schwerpunkt auf Fabry-Krankheit und Pompe-Krankheit liegt.
Die Präsentationen umfassen drei Poster zur Fabry-Krankheit, die über die von Patienten berichteten Ergebnisse des FollowME Fabry Pathfinders-Registers, die Lebensqualität von mit Migalastat behandelten Jugendlichen und den Verlauf von Patienten, die in Polen mit der Fabry-Krankheit leben, diskutieren. Zwei Poster zur Pompe-Krankheit werden die Ergebnisse nach der Basislinie des britischen Early Access to Medicines Scheme-Registers für Cipaglucosidase Alfa zusammen mit Miglustat und Miglustat als erstklassigen Enzymstabilisator für spät einsetzende Pompe-Krankheit abdecken.
- Amicus Therapeutics is presenting five posters at a major international symposium, demonstrating ongoing research and development efforts
- The company's presentations cover both Fabry Disease and Pompe Disease, showing a diverse research portfolio
- Participation in the symposium may increase visibility and credibility in the rare disease research community
- None.
PRINCETON, N.J., Sept. 03, 2024 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that five posters across its development programs will be included at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium, being held September 3-6, 2024 in Porto, Portugal.
Poster Sessions:
Fabry Disease:
Abstract Title: FollowME Fabry Pathfinders registry: patient-reported outcomes in a cohort of patients on migalastat treatment for at least two years (Poster #21180)
- Presenter: Aleš Linhart, D.Sc., Charles University and General University Hospital in Prague, Prague, Czech Republic
- Poster Session: Wednesday, September 4, 6:15 – 8:15 p.m. WET
- Location: Poster Room, Rosa Mota Pavilion
Abstract Title: Quality of life of migalastat-treated adolescents with Fabry disease: results from the ASPIRE study and open-label extension (Poster #21202)
- Presenter: Amarilis Sanchez-Valle, MD, University of South Florida Health, Tampa, U.S.A.
- Poster Session: Wednesday, September 4, 6:15 – 8:15 p.m. WET
- Location: Poster Room, Rosa Mota Pavilion
Abstract Title: Exploring the journey of patients living with Fabry disease in Poland (Poster #20789)
- Presenter: Anna Moskal, The Association of Families with Fabry Disease, Wroclaw, Poland
- Poster Session: Wednesday, September 4, 6:15 – 8:15 p.m. WET
- Location: Poster Room, Rosa Mota Pavilion
Pompe Disease:
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 #20863)
- Presenter: Elaine Murphy, MD, University College London Hospitals, London, U.K.
- Poster Session: Wednesday, September 4, 6:15 – 8:15 p.m. WET
- Guided Poster Walk: Thursday, September 5, 12:10 – 1:40 p.m. WET
- Location: Poster Room, Rosa Mota Pavilion
Abstract Title: Miglustat: a first-in-class enzyme stabilizer for late-onset Pompe disease (Poster #20873)
- Presenter: Mark Roberts, MD, Salford Royal NHS Foundation Trust, Greater Manchester Neurosciences Centre, Manchester, U.K.
- Poster Session: Wednesday, September 4, 6:15 – 8:15 p.m. WET
- Location: Poster Room, Rosa Mota Pavilion
For more information on the SSIEM 2024 Annual Symposium, please visit ssiem2024.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
Head of Global Corporate Communications
dmoore@amicusrx.com
(609) 662-5079
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