Full Press Release Details
PHILADELPHIA, Feb. 07, 2022 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced that two oral presentations and 11 posters across its development programs will be included at the 18th Annual WORLDSymposium™ 2022, being held February 7-11, 2022 in San Diego, CA.
Oral Platform Presentation:
Wednesday, February 9, 10:30 a.m. PST
Abstract Title: Immunogenicity of cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease (LOPD): A phase III, randomized study (PROPEL)
Presenter: Tahseen Mozaffar, MD, University of California, Irvine, CA, U.S.A.
Location: Exhibit Hall
Friday, February 11, 10:15 a.m. PST
Abstract Title: Liver-directed and systemic AAV gene transfer approaches for Pompe disease therapy
Presenter: Naresh Kumar Meena, PhD, National Institutes of Health, Bethesda, MD, U.S.A.
Location: Exhibit Hall
Wednesday, February 9, 3:00-5:00 p.m. PST
Abstract Title: Baseline demographics and clinical characteristics of patients enrolled in the followME Fabry Pathfinders Registry (Poster #216)
Presenter: Peter Nordbeck, MD, University Hospital Wuerzburg, Wuerzburg, Germany
Location: Exhibit Hall
Abstract Title: Long-term multisystemic efficacy with migalastat in ERT-naive and ERT-experienced patients with amenable GLA variants (Poster #123)
Presenter: Robert Hopkin, MD, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, U.S.A.
Location: Exhibit Hall
Abstract Title: Migalastat HCl 150 mg every other day is well-tolerated and efficacious in adolescent patients with Fabry disease (Poster #258)
Presenter: Uma Ramaswami, MD, Royal Free London NHS Foundation Trust, London, U.K.
Location: Exhibit Hall
Abstract Title: Insights into patients’ expectations and treatment preferences based on the patient needs questionnaire: Interim results from the SATIS-Fab study in Fabry disease (ePoster #174)
Presenter: Olivier Lidove, MD, Deaconess Hospital Group Croix Saint-Simon, Paris, France
Location: Virtual Site
Thursday, February 10, 3:00-5:00 p.m. PST
Abstract Title: First results from The Spanish Fabry Women Study: A retrospective observational study describing the phenotype of female carrying genetic variants associated to Fabry disease (ePoster #269)
Presenter: Rosario Sánchez, MD, General University Hospital of Alicante, Alicante, Spain
Location: Virtual Site
Abstract Title: Fabry disease, symptom burden, health-related quality of life burden and treatment satisfaction (ePoster #186)
Presenter: Alasdair MacCulloch, PhD, Amicus Therapeutics UK LTD, Marlow, U.K.
Location: Virtual Site
Abstract Title: Treatment preferences in Fabry disease, a discrete choice experiment in the UK and Denmark (ePoster #187)
Presenter: Alasdair MacCulloch, PhD, Amicus Therapeutics UK LTD, Marlow, U.K.
Location: Virtual Site
Wednesday, February 9, 3:00-5:00 p.m. PST
Abstract Title: Immunogenicity of cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease (LOPD): A phase III, randomized study (PROPEL) (Poster #47)
Presenter: Tahseen Mozaffar, MD, University of California, Irvine, CA, USA
Location: Exhibit Hall
Abstract Title: Cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease (LOPD): PROPEL study subgroup analyses (Poster #39)
Presenter: Barry Byrne, MD, PhD, University of Florida Health, Gainesville, FL, U.S.A.
Location: Exhibit Hall
Abstract Title: Living with Pompe disease in the UK: Characterizing the patient journey and burden on physical, emotional and social quality of life (ePoster #130)
Presenter: Derralynn Hughes, BMBCh, University College London, London, U.K.
Location: Virtual Site
Thursday, February 10, 3:00-5:00 p.m. PST
Abstract Title: Plasma total GAA protein PK profiles differ between cipaglucosidase alfa/miglustat and alglucosidase alfa (ePoster #143)
Presenter: Franklin Johnson, MS, Amicus Therapeutics, Inc., Philadelphia, PA, U.S.A.
Location: Virtual Site
About WORLDSymposium
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
Executive Director, Investor Relations
Head of Global Corporate Communications