CRANBURY, N.J., Aug. 27, 2012 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of developing therapies for rare and orphan diseases, today announced that 1 presentation and 4 posters related to its investigational pharmacological chaperones will be included at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2012. SSIEM 2012 will be held September 4-7, 2012 in Birmingham, United Kingdom.
An Ongoing Phase 2a Study to Investigate the Effects of AT2220 (duvoglustat HCl) on the Pharmacokinetics of Acid Alpha-Glucosidase in Subjects with Pompe Disease - Preliminary Results. - Kishnani P., Tarnopolsky M., Sivakumar K., Byrne B., Goker-Alpan O., Guter K., Pervaiz M., Dasouki M., Levine T., Roberts M., Johnson F., Sitaraman S., Winkler R., Khanna R., Flanagan J., Sjoberg E., Valenzano K., Lockhart D., Boudes P.
- Poster: September 5, 2012, 17:30 – 19:30 BST; September 6, 2012 12:00 -13:00 BST
A Novel Phase 2a Study Design to Investigate the Effect of AT2220 (duvoglustat HCl) on the Pharmacokinetics of Acid Alpha-Glucosidase in Subjects with Pompe Disease. – Kishnani P., Tarnopolsky M., Sivakumar K., Byrne B., Goker-Alpan O., Guter K., Pervaiz M., Dasouki M., Levine T., Roberts M., Johnson F., Winkler R., Valenzano K., Lockhart D., Boudes P.
- Poster: September 5, 2012, 17:30 – 19:30 BST; September 6, 2012 12:00 -13:00 BST
The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid Alpha-Glucosidase Tissue Uptake and Improves Glycogen Reduction in a Mouse Model of Pompe Disease. – Pellegrino L., Feng J., Frascella M., Lun Y., Flanagan J., Guillen D., Benjamin E., Lockhart D., Valenzano K., Khanna R.
- Poster: September 5, 2012, 17:30 – 19:30 BST; September 6, 2012 12:00 -13:00 BST
Migalastat HCl, an Investigational Pharmacological Chaperone Therapy: Screening Results from FACETS, a Phase 3 Study in Male and Female Fabry Patients. – Nicholls, K., Castelli J., Winkler, Sitaraman, S., Benjamin E., Wu, X., Duke C., Boudes, P., on Behalf of FACETS Study AT1001-011 Principal Investigators.
- Poster: September 5, 2012, 17:30 – 19:30 BST; September 6, 2012 12:00 -13:00 BST
Migalastat HCl Reduces Globtriaosylsphingosine (Lyso-GB3) in Plasma of Fabry Patients and in Tissues of Fabry Transgenic Mice. – Benjamin E., Brignol N., Young B., Chang H., Khanna R., Soska R., Sitaraman S., Germain D., Giugliani R., Hughes D., Mehta A., Nicholls K., Boudes P., Lockhart D., Valenzano K.
- Presentation: September 6, 2012, 13:00 – 14:30 BST
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of developing therapies for rare and orphan diseases. The Company is developing orally-administered, small molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of human genetic diseases. Amicus' late-stage programs for lysosomal storage disorders include migalastat HCl monotherapy in Phase 3 for Fabry disease; migalastat HCl co-administered with enzyme replacement therapy (ERT) in Phase 2 for Fabry disease; and AT2220 co-administered with ERT in Phase 2 for Pompe disease.
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