April 15, 2021 will be our 8^th Annual International Pompe Day!

The IPA believes that raising awareness of Pompe Disease is key, and this year’s theme has been selected as “Pompe Around the World.”

How You can Participate:

1. Our goal with Pompe Around the World is to show what it is like to live, cope, or how you are connected with Pompe all over the world — that no matter where we live we are all connected. Please take a picture that represents where you live and share a short statement about your life.
2. Send an email to ipd2021@worldpompe.org

• the subject of your email will be the title of your post;
• the body of the email will be the post itself, where you can share your short statement;
• to include your photo in your post, you can simply attach the image to your email (total size max. 10 MB).
• Please include your name, country and age in the email, so we can show the world how strong our Community is.

By submitting a contribution, you agree that it is published on our 2021 International Pompe Day blog: ipompeday.blogspot.com

The Blog will go live on International Pompe Day—April 15, 2021. However, we will post a list of submissions, which will be periodically updated, so that you can see if your submission has been received.

Thank you!

PHILADELPHIA, Feb. 11, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics, a patient-dedicated global biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases, today announced the topline results of its Phase 3 PROPEL Pivotal Trial for AT-GAA (cipaglucosidase alfa and miglustat), its investigational two-component therapy for the treatment of late-onset Pompe disease (LOPD) that has previously received Breakthrough Therapy Designation from the U.S. FDA and the Promising Innovative Medicine designation from the MHRA in the United Kingdom. With consent from the FDA, the Company initiated a rolling Biologics License Application (BLA) in the fourth quarter of 2020. Amicus plans to complete the BLA submission in the second quarter of this year and anticipates additional regulatory submissions in the European Union and in other geographies throughout 2021.

To read more click here.

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.

Spark Therapeutics has announced that the first participant has been dosed in phase 1/2 study of investigational gene therapy for Late-Onset Pompe Disease.

To read the full press release click here: https://sparktx.com/press_releases/first-patient-dosed-pompe/

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.