CRANBURY, N.J., Jan. 26, 2015 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of rare and orphan diseases, today announced that 2 oral presentations and 6 posters highlighting its development programs for lysosomal storage diseases will be included at the 11th Annual Lysosomal Disease Network WORLD Symposium (LDN WORLD), to be held February 9-13, 2015 in Orlando, FL.
Oral Platform Presentations:
Fabry Disease:
- Long-Term Efficacy and Safety of Migalastat Compared to Enzyme Replacement Therapy in Fabry Disease: Phase 3 Study Results – Derralynn Hughes, DPhil, University College London (Thursday, February 12 at 9:45 a.m. ET)
Pompe Disease:
- Novel Recombinant Human Acid Alpha-Glucosidase with Optimal Glycosylation is Significantly Better than Standard of Care Enzyme Replacement for Glycogen Clearance in Skeletal Muscles of Gaa Knock-out Mice. – Russell Gotschall, Amicus Therapeutics, Inc. (Tuesday, February 10 at 4:15 p.m. ET)
Posters: Tuesday, February 10 - Thursday, February 12, 4:30-6:30 p.m. ET
Fabry Disease:
- Long-Term Efficacy and Safety of Migalastat Compared to Enzyme Replacement Therapy in Fabry Disease: Phase 3 Study Results – Derralynn Hughes, DPhil, University College London (Poster #115)
- Migalastat Reduces Left Ventricular Mass Index in Fabry Patients Naïve to ERT and Previously Treated with ERT – Daniel Bichet, M.D., M.Sc., University of Montreal (Poster #LB-2 – Late Breaker)
- Improvement in Gastrointestinal Symptoms Observed in the Phase 3 FACETS Study of Migalastat in Fabry Patients – Raphael Schiffmann, M.D., M.H.Sc., Baylor University (Poster #230)
- Accurate Quantitation of Plasma Globotriaosylsphingosine (lyso-Gb3) in Normal Individuals and Fabry Patients by Liquid Chromatography-Tandem Mass Spectrometry (LC-MS/MS) – Rick Hamler, Amicus Therapeutics, Inc. (Poster #100)
Pompe Disease:
- Novel Recombinant Human Acid Alpha-Glucosidase with Optimal Glycosylation is Significantly Better than Standard of Care Enzyme Replacement for Glycogen Clearance in Skeletal Muscles of Gaa Knock-out Mice. – Russell Gotschall, Amicus Therapeutics, Inc. (Poster #94)
- Histological Examination of the Effect of a Highly Phosphorylated Proprietary Recombinant Human Acid Alpha-Glucosidase on Glycogen Reduction in Disease-Relevant Muscles of Pompe Mice – Yi Lun, Amicus Therapeutics, Inc. (Poster #162)
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of therapies for rare and orphan diseases. The Company is developing novel, first-in-class treatments for a broad range of human genetic diseases, with a focus on delivering new benefits to individuals with lysosomal storage diseases. Amicus' lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, as well as next-generation enzyme replacement therapy (ERT) products for Fabry disease, Pompe disease, and MPS-1.
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