Welcome to the International Pompe Association (IPA)
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.
The New Zealand Organisation for Rare Disorders (NZORD) will present the New Zealand Rare Disease Day Awards (RDD Awards) for the first time in 2019. The winner in the Patient Advocacy and Support Award category is Allyson Lock. She has been nominated as a tireless fighter for the Pompe community.
Allyson has spent the past eight years lobbying and supporting patients in New Zealand. She is President of the New Zealand Pompe Network and since her diagnosis has fought to ensure that the cost of treating Pompe with enzyme replacement therapy is covered by the New Zealand health care system.
We congratulate Allyson on the award.
(Photo courtesy of Allyson Lock and the New Zealand Organisation for Rare Disorders, photographed by Levi Gershkowitz of Living In The Light Of Rare Diseases.)
The 2019 IPA calendar is finally out and has been distributed to member associations worldwide. We thank the International Pompe Community that contributed to the creation of a calendar that will keep Pompe in the minds and hearts of all recipients throughout the year. The calendar will give us the opportunity to raise awareness on Pompe disease, for not just a day, but for the whole year of 2019, which marks also the IPA's 20th anniversary.
The calendar features for each month photos and artworks by the Pompe community, in addition to thought-provoking statements or questions by and for the scientific community.
The International Pompe Association.
- 2019 IPA Calendar (PDF, 4,04 MB)