April 15, 2019

Today is our 6^th International Pompe Day!

The goal of International Pompe Day is to foster international awareness of Pompe Disease and the slogan for our day is Together We Are Strong.

The IPA believes that raising awareness about Pompe Disease is key, and for 2019 the theme that has been selected is Moving On with Pompe: 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, in the hope of inspiring others.

Inspiring stories from our international community have been collected in a dedicated blog that the IPA has specifically created: https://ipompeday.blogspot.com

Please read and share these stories, so we can inspire others and show the world how strong our Community is!

Thanks to all that have contributed with their story, and happy International Pompe Day!

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

Find more
(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.

Read more

• 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.