Amicus Therapeutics Announces Participation at the SSIEM 2024 Annual Symposium

Rhea-AI Summary

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.

Positive

• 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

Negative

• 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

FOLD–G

FAQ

What is Amicus Therapeutics presenting at the SSIEM 2024 Annual Symposium?

Amicus Therapeutics (FOLD) is presenting five posters at the SSIEM 2024 Annual Symposium, covering research on Fabry Disease and Pompe Disease. The posters include patient-reported outcomes, quality of life studies, and clinical trial results for treatments like migalastat and cipaglucosidase alfa plus miglustat.

When and where is the SSIEM 2024 Annual Symposium taking place?

The Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium is being held from September 3-6, 2024, in Porto, Portugal.

What specific topics are covered in Amicus Therapeutics' (FOLD) Fabry Disease posters?

Amicus Therapeutics' Fabry Disease posters cover patient-reported outcomes from the FollowME Fabry Pathfinders registry, quality of life in migalastat-treated adolescents from the ASPIRE study, and the journey of patients living with Fabry disease in Poland.

What are the main topics of Amicus Therapeutics' (FOLD) Pompe Disease presentations?

Amicus Therapeutics' Pompe Disease presentations focus on post-baseline outcomes of the UK Early Access to Medicines Scheme registry for cipaglucosidase alfa plus miglustat in late-onset Pompe disease, and miglustat as a first-in-class enzyme stabilizer for late-onset Pompe disease.