Amicus Therapeutics Announces Presentations and Posters at the 20th Annual WORLDSymposium™ 2024
01 Febrero 2024 - 6:00AM
Amicus Therapeutics (Nasdaq: FOLD), today announced that one oral
presentation and 11 posters across its development programs will be
included at the 20th Annual WORLDSymposium™ 2024, being held
February 4-9, 2024 in San Diego, CA.
Oral Platform Presentation:
Fabry Disease:
Abstract Title: Trial in
progress: An open-label study (AT1001-025) to evaluate the safety
and pharmacokinetics of migalastat in patients with Fabry disease
and amenable GLA variants and severe renal impairment or end-stage
renal disease treated with hemodialysis
- Presenter:
Franklin Johnson, MS, Amicus Therapeutics, Inc., Princeton, NJ,
U.S.A.
- Date and
time: Thursday, February 8, 9:12 – 9:24 a.m. PT
Poster Sessions:
Fabry Disease:
Abstract Title: Multiorgan
involvement in females with Fabry disease: Results from two Phase
III trials and the followME registry (Poster #246)
- Presenter: Peter
Nordbeck, MD, University Hospital Würzburg, Würzburg, Germany
- Date and time:
Wednesday, February 7, 3:00 – 5:00 p.m. PT
- Location: Exhibit
Hall, Kiosk 41-F
Abstract Title: Prevalence of
migalastat-amenable mutations in patients with Fabry disease from
Brazil, Argentina and Colombia (Poster #262)
- Presenter: Juan
Politei, MD, Foundation for the Study of Neurometabolic Diseases,
Buenos Aires, Argentina
- Date and time:
Wednesday, February 7, 3:00 – 5:00 p.m. PT
- Location: Exhibit
Hall, Kiosk 44-F
Abstract Title:
Treatment-related benefit and satisfaction in patients with Fabry
disease: Insight into patients’ expectations and preferences from
the SATIS-Fab study (Poster #189)
- Presenter: Didier
Lacombe, MD, Bordeaux Hospital University Center, Bordeaux,
France
- Date and time:
Wednesday, February 7, 3:00 – 5:00 p.m. PT
- Location: Exhibit
Hall, Kiosk 34-B
Abstract Title: FollowME Fabry
Pathfinders registry: Renal effectiveness in a cohort of patients
on migalastat treatment for at least three years (Poster #339)
- Presenter: Michael
West, MD, Dalhousie University, Halifax, Canada
- Date and time:
Wednesday, February 7, 3:00 – 5:00 p.m. PT
- Location: Exhibit
Hall, Kiosk 49-D
Abstract Title: Exploring the
experience of females living with Fabry disease in North
America (Poster #8)
- Presenter: Julia
Alton, Executive Director, Canadian Fabry Association, Ontario,
Canada
- Date and time:
Wednesday, February 7, 3:00 – 5:00 p.m. PT
- Location: Exhibit
Hall, Kiosk 2-F
Abstract Title: Trial in
progress: An open-label study (AT1001-025) to evaluate the safety
and pharmacokinetics of migalastat in patients with Fabry disease
and amenable GLA variants and severe renal impairment or end-stage
renal disease treated with hemodialysis (Poster #165)
- Presenter:
Franklin Johnson, MS, Amicus Therapeutics, Inc., Princeton, NJ,
U.S.A.
- Date and time:
Thursday, February 8, 3:00 – 5:00 p.m. PT
- Location: Exhibit
Hall, Kiosk 18-D
Abstract Title: Cardiac
biomarkers in Fabry disease (Poster #241)
- Presenter: Pooja
Nandi, Scientific Product Manager, Koneksa Health, New York, NY,
U.S.A.
- Date and time:
Thursday, February 8, 3:00 – 5:00 p.m. PT
- Location: Exhibit
Hall, Kiosk 25-D
Abstract Title: Exploring the
journey of patients with Fabry disease in Brazil (Poster
#346)
- Presenter:
Christopher Wingrove, Amicus Therapeutics UK LTD, Marlow, U.K.
- Date and time:
Thursday, February 8, 3:00 –5:00 p.m. PT
- Location: Exhibit
Hall, Kiosk 31-H
Pompe Disease:
Abstract Title: Effect size
analysis of cipaglucosidase alfa plus miglustat versus
alglucosidase alfa in ERT-experienced adults with late-onset Pompe
disease in PROPEL (Poster #232)
- Presenter: Tahseen
Mozaffar, MD, University of California, Irvine, CA, U.S.A.
- Date and time:
Wednesday, February 7, 3:00 – 5:00 p.m. PT
- Location: Exhibit
Hall, Kiosk 39-B
Abstract Title: Switching
treatment to cipaglucosidase alfa plus miglustat positively affects
motor function and quality of life in patients with late-onset
Pompe disease (Poster #320)
- Presenter: Antonio
Toscano, MD, University of Messina, Messina, Italy
- Date and time:
Wednesday, February 7, 3:00 – 5:00 p.m. PT
- Location: Exhibit
Hall, Kiosk 45-D
Abstract Title Baseline
demographics of the UK Early Access to Medicines Scheme registry
for cipaglucosidase alfa plus miglustat in enzyme replacement
therapy-experienced adults with late-onset Pompe disease (Poster
#279)
- Presenter: Mark
Roberts, MD, Salford Royal NHS Foundation Trust, Greater Manchester
Neurosciences Centre, Manchester, U.K.
- Date and time:
Wednesday, February 7, 3:00 – 5:00 p.m. PT
- Location: Exhibit
Hall, Kiosk 46-F
Abstract Title: Minimal
clinically important differences in six-minute walking distance in
late-onset Pompe disease (Poster #293)
- Presenter:
Benedikt Schoser, MD, Ludwig Maximilian University of Munich,
Munich, Germany
- Date and time:
Wednesday, February 7, 3:00 – 5:00 p.m. PT
- Location: Exhibit
Hall, Kiosk 50-F
About
WORLDSymposiumWORLDSymposium™ is an annual research
conference dedicated to lysosomal diseases. WORLD is an acronym
that stands for We’re Organizing Research on Lysosomal Diseases.
The goal of WORLDSymposium is to provide an interdisciplinary forum
to explore and discuss specific areas of interest, research and
clinical applicability related to lysosomal diseases. Each year,
WORLDSymposium hosts a scientific meeting presenting the latest
information from basic science, translational research, and
clinical trials for lysosomal diseases. This symposium is designed
to help researchers and clinicians to better manage and understand
diagnostic options for patients with lysosomal diseases, identify
areas requiring additional basic and clinical research, public
policy and regulatory attention, and identify the latest findings
in the natural history of 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 Twitter 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 Communicationsdmoore@amicusrx.com(609)
662-5079
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