New Zealand Rare Disease Day Award 2019 for Allyson Lock

The New Zealand Organisation for Rare Disorders (NZORD) will present the New Zealand Rare Disease Day Awards (RDD Awards) for...
Author: Webmaster
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December 18, 2018

2019 IPA Awareness Calendar

  The 2019 IPA calendar is finally out and has been distributed to member associations worldwide. We thank...
Author: Fabio Di Pietro
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December 10, 2018

Community Advisory Board (CAB) – Call for Applications

The IPA is establishing a Community Advisory Board (CAB) for Pompe disease. A CAB is a group of...
Author: IPA
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September 24, 2018

Enzyme Replacement Therapy Reduces The Risk For Wheelchair Dependency In Adult Pompe Patients

We would like to share the latest publication based on the data generated through the IPA/ Erasmus MC Pompe...
Author: IPA
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May 30, 2018

2019 IPA Calendar: Call for Submissions

At the International Pompe Association’s Annual General Meeting in Nottingham, the IPA Board and Members who were present...
Author: Tiffany House
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April 14, 2018

The Humanistic Burden of Pompe Disease: Are There Still Unmet Needs? A Systematic Review.

Dear Pompe Patient Community Leaders, At Audentes Therapeutics, we are focused on developing and commercializing gene therapy products...
Author: Audentes
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January 25, 2018

Update on a Clinical Trial (COMET Study by Sanofi Genzyme)

NeoGAA, a new generation of enzyme replacement therapy developed by Sanofi Genzyme will be tested in 96 patients...
Author: Sanofi Genzyme
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September 29, 2017

Obituary to Henri Termeer

The IPA Board was extremely saddened to learn of the unexpected passing of Henri Termeer, former CEO of...
Author: IPA
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May 16, 2017

Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Pompe Phase 1/2 Study

CRANBURY, N.J., May 15, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD) today announced positive functional data from initial...
Author: Amicus
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May 14, 2017

Living Healthy with Pompe

Tips and Tricks from the Pompe Community April 15, 2017 is the Fourth Annual International Pompe Day. The...
Author: Webmaster
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April 14, 2017
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