Welcome to the International Pompe Association (IPA)
Moving On with Pompe!
April 15, 2019 will be our 6th International Pompe Day!
The IPA believes that raising awareness about Pompe Disease is key, and for this year the theme that has been selected is “Moving On with Pompe”: This is meant to be an encouraging and positive theme to show how it is possible to continue living a fulfilling life after diagnosis and despite the challenges of living with Pompe disease.
The International Pompe Association is calling on our global Pompe community to show how every one of us is Moving On with Pompe in the hope of inspiring others.
In order to collect these stories, the IPA has created a blog that individuals can post to.
How You can Participate:
You can share your photos and story, and inspire others on how you are Moving On with Pompe, by sending an email to [submission is closed].
- the subject of your email will be the title of your post;
- the body of the email will be the post itself;
- to include a photo in your post, you can simply attach an 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 2019 International Pompe Day blog: https://ipompeday.blogspot.com/
Your submissions are not published before International Pompe Day on April 15, 2019, but we will post a list of submissions which is updated from time to time.
There are multiple clinical trials that are currently underway for Pompe disease. Because there are so many different trials, which is unique for a rare disease like Pompe, it is important to understand the clinical trial process, the trials phases, and other basic information.
Please find in the attachment a short overview of clinical trials in general.
The International Pompe Association
(courtesy of the AMDA - Acid Maltase Deficiency Association)
- Overview of Clinical Trials (PDF, 320.97 kB)
CRANBURY, N.J., Dec. 20, 2018 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, today announced the dosing of the first patient in a global phase 3 clinical study (ATB200-03, or PROPEL) of AT-GAA in adult patients with late onset Pompe disease. PROPEL is a 52-week, double-blind randomized study designed to assess the efficacy, safety and tolerability of AT-GAA compared to the current standard of care, alglucosidase alfa, an enzyme replacement therapy (ERT).
All participants randomized to AT-GAA in the PROPEL study will receive drug manufactured at the 1000L scale intended for clinical and commercial supply. Amicus also expects to initiate a smaller, open-label study of AT-GAA in pediatric patients in 2019.
“The initiation of our global PROPEL study is a true example of our capabilities to discover, develop and manufacture promising therapies, and deliver them to patients as quickly as we can,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc.
- Press release (external link)
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.