News

Cover - Approval

Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the European Union

Amicus Therapeutics announces that the European Commission (EC) has granted approval for Opfolda® (miglustat), an enzyme stabilizer of cipaglucosidase alfa, an...
Author: IPA
News
June 28, 2023

Patient Registries for Pompe Disease Survey

The IPA invites you to share your perspective about Patient Registries for Pompe disease.
Author: IPA
News
June 4, 2023
International Pompe Day-2023

2023: Every Move Counts

The IPA is excited to announce its plans for International Pompe Day. This year, we are focusing on raising awareness...
Author: IPA
News
May 17, 2023

Pompe Patients Covid Survey

Over the last 2 years, it is clear that the COVID-19 pandemic has had wide reaching effects on people all...
Author: IPA
Archive
September 19, 2022
International Pompe Day 2022

International Pompe Day 15 April 2022

International Pompe Voices   For International Pompe Day (IPD) we are asking people in the global Pompe community to record...
Author: IPA
Archive
March 21, 2022
Cover - Ukraine

International Pompe Association supports Ukrainian Pompe patients

Author: Maryze Schoneveld van der Linde, Volunteer, Communications Coordinator, International Pompe Association Date: March 19, 2022 On February 24, 2022, at...
Author: IPA
Archive
March 20, 2022
Cover - documents - reports

IPA Position Paper on Patient Reported Outcomes

One common challenge that all potential future therapies will face is how to best design trials in an ultra-raredisease with...
Author: Webmaster
Archive
November 22, 2021
Cover-webinar

WORLDSymposium 2021 Webcast

The WORLDSymposium is an annual conference which brings together clinicians, scientists, industry and patients from all over the world.
Author: IPA
Archive
March 22, 2021
International Pompe Day 2021

2021: Pompe Around the World

This year’s theme is “Pompe Around the World.” No matter where we live we are all connected.
Author: IPA
Archive
February 22, 2021
Cover-news-clinical-trial

Amicus Announces PROPEL Phase 3 Study Topline Results

PHILADELPHIA, Feb. 11, 2021 (GLOBE NEWSWIRE) — Amicus Therapeutics, a patient-dedicated global biotechnology company focused on discovering, developing and delivering...
Author: Amicus
Archive
February 12, 2021
News - clinical trial - cover

Update from Spark Therapeutics

Spark Therapeutics has announced that the first participant has been dosed in phase 1/2 study of investigational gene therapy for...
Author: Spark Therapeutics
Archive
February 1, 2021
Cover-news-clinical-trial-covid

Update from Spark Therapeutics

In March 2020, after consultation with global investigators, advocacy leadership, and healthcare experts, and in an abundance of caution for...
Author: Spark Therapeutics
Archive
August 20, 2020