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Amicus Therapeutics Presents Additional Positive Preliminary Phase 1/2 Data

| Source: Amicus | Print

CRANBURY, N.J. and SAN DIEGO, Feb. 15, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD)... today presented additional positive preliminary data from a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in a poster at the 13th Annual WORLDSymposium™ in San Diego, CA. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone.

"We are very pleased to share these additional positive preliminary clinical results here at the WORLDSymposium as we continue with the cascade of data from this important study in Pompe Disease patients," said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. "In addition to excellent continued safety, tolerability and PK profile, we now see evidence that this novel Amicus treatment paradigm is further reducing important biomarkers of disease in both ERT-switch patients as well as in the first ERT-treatment naïve patients.  In particular, demonstrating with these data that we can further reduce these biomarkers in a switch population is very encouraging. To our knowledge, there are no published data for other Pompe ERTs where these types of reductions in biomarkers have been shown. Moreover, the fact that these biomarkers have demonstrated a sustained reduction in a majority of patients over the first 18 weeks suggests that the effects of our approach may indeed be persistent and durable. We look forward to data in more patients and over longer periods of time in the months ahead, including important measures of muscle function in these patients. We could not be more pleased with the preliminary data to date."...

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Disclaimer: The IPA does not endorse any of the products, medications, treatments or information reported herein. Articles on the IPA web pages are intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.

Amicus Therapeutics Presents Scientific Findings and Preclinical Data for Pompe Program

| Source: Amicus | Print

CRANBURY, N.J. and SAN DIEGO, Feb. 15, 2017 (GLOBE NEWSWIRE) --  Amicus Therapeutics (Nasdaq:FOLD)... today presented new scientific findings and preclinical data on functional outcomes in an oral presentation and poster at the 13th Annual WORLDSymposium™ in San Diego, CA. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake in muscles, co-administered with AT2221, a pharmacological chaperone designed to stabilize ERT in circulation. 

In previously reported preclinical studies, ATB200 was associated with increased tissue enzyme levels and reduced glycogen levels in muscle, which was further improved when AT2221 was co-administered with ATB200. For the first time at WORLDSymposium, Hung Do, PhD, Chief Science Officer of Amicus Therapeutics, presented results from preclinical studies that showed ATB200/AT2221 reversed cellular dysfunction and progressively increased muscle strength following every-other-week administration at 20 mg/kg over a five month period in an animal model of Pompe disease (GAA knock-out mice)...

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Disclaimer: The IPA does not endorse any of the products, medications, treatments or information reported herein. Articles on the IPA web pages are intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.

Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of Novel Treatment Paradigm for Pompe Disease

| Source: Amicus | Print

Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of  Novel Treatment Paradigm for Pompe Disease

CRANBURY, N.J., Dec. 08, 2016 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today announced positive preliminary data from a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone...

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Disclaimer: The IPA does not endorse any of the products, medications, treatments or information reported herein. Articles on the IPA web pages are intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.

  1. 10 Years of Enzyme Replacement Therapy
  2. NeoGAA Phase 3 Trial Begins
  3. Statement from IPA on Biomarin
  4. BioMarin Update for the Pompe Community
  5. IPA Statement on Biomarin's June 9th announcement it will seek to out-license BMN-701
  6. The Walker Society – a beneficiary research fund in honour of Helen Walker
  7. 2016 International Pompe Day Art Contest: Results
  8. Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease
  9. BioMarin Announces Interim Analysis of INSPIRE Clinical Trial in Pompe Disease at 34th Annual J.P. Morgan Annual Healthcare Conference
  10. Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease
  11. The Swiss Pompe Group
  12. 2015 AMDA International Pompe Patient and Scientific Conference - Summary Report
  13. Pompe Registry - A Decade of Making a Difference
  14. #AMDAIPA Pompe Conference on Twitter
  15. Australia has admitted life-saving drug for Pompe disease in the Australian Life-Saving Drug Programme
  16. Visit to the World War II Commemoration Monument in Saint Pancras (The Netherlands)
  17. Registration is now open for the 2015 International Pompe Patient and Scientific Conference
  18. Genzyme Pompe Community Program Update
  19. Amicus Therapeutics Honors Fabry Disease Awareness Month and International Pompe Day
  20. Poll: IPD Fundraiser Ideas 2015
  21. Abstracts from the 7th European Symposium on Steps Forward in Pompe Disease 2014
  22. Australia Approves Treatment for Young Australians with Pompe Disease
  23. Amicus Therapeutics Presents Preclinical Data on Next-Generation Pompe ERT at WORLDSymposium(TM) 2015
  24. Call for Participation in the Pompe (Em)Power Project
  25. A Wonderful Fund Raising 'Every Life Counts' Charity Dinner Graced by Negeri Sembilan's King and Queen
  26. IPA Statement and Clarification on Potential New Production Method Mentioned in Dutch Interview
  27. Australian Pompe’s Association held its first National patient and family support forum in Melbourne
  28. ENMC workshop on Pompe Disease
  29. International Pompe Day Commemorative Book Now Available!
  30. IPA's Pompe M-PowerPilot Project Recipient of Genzyme's 4th Annual PAL Award
  31. Donation Received from Turkish Charity Fund
  32. Expression of Hope III
  33. New Chairwoman for International Pompe Association
  34. Resounding Success International Pompe Day 2014
  35. Carine van Capelle receives PhD with her thesis of Children with Pompe disease: clinical characteristics, peculiar features and effects of enzyme replacement therapy
  36. Linda van den Berg receives PhD with her thesis on 'The Musculoskeletal System in Pompe Disease - Pathology, consequences and treatment options'
  37. Juna de Vries receives PhD on Natural course, effects of enzyme therapy and prognostic factors in adults with Pompe disease
  38. First tree planted in Pompe Garden in Warsaw
  39. Attention for Pompe Disease in Russian newspaper
  40. Hero Award for Malaysian LSD Association President Ir. YeeSeng Lee
  41. Raising Awareness with social media on International Pompe Day in Australia
  42. Poland and Italy celebrate International Pompe Day
  43. International Pompe Day 2014
  44. Soccer Team RCD Espanyol supports International Pompe Day
  45. Colombian LSD Association (ACOPEL) participates in International Pompe Day

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