One common challenge that all potential future therapies will face is how to best design trials in an ultra-raredisease with a heterogenous late-onset population. On behalf of the international Pompe community, the IPA would like to take this opportunity to advocate for an increased value to be placed on Patient Reported Outcomes in clinicaltrial design, as well as in regulatoryreview of future treatment option.

Due to the rare nature of Pompe, which leads to the necessity of small trial sizes and the heterogenous nature of Pompe disease, patients in trials are likely to be at different stage in the development of their disease. Furthermore, just as there is a great degree of heterogeneity in Pompe disease progression for patients, over the last twenty years we have seen that there is also a large degree of heterogeneity in patient response to treatment. Therefore, we believe the broadest range of evaluation is necessary to ensure that all the benefits of treatment are seen. And with a progressive disease like Pompe, we must not forget that stabilization of symptoms is a benefit for patients.

Read the full IPA Position Paper on Patient Reported Outcomes (PDF, 925 kB)

April 15, 2021

Today is our 8^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 the theme selected for 2021 is Pompe Around The World in order to show what it is like to live, cope, or how we are connected with Pompe all over the world, that no matter where we live we are all connected as a united community.

Our Pompe Around the World contributions have been collected in a dedicated blog that the IPA has specifically created ipompeday.blogspot.com/2021

Thanks to all that have contributed, and happy International Pompe Day to our amazing Pompe community, to the Pompe warriors, families, carers, medical specialists and all those contributing to a better future all around the world!

#TogetherWeAreStrong #InternationalPompeDay #PompeAroundTheWorld

The WORLD symposium is an annual conference which brings together clinicians, scientists, industry and patients from all over the world to learn and share knowledge on all Lysosomal Diseases (LDs). In consideration of the current COVID-19 pandemic, this year the conference was virtual-only.

Please join us in this webcast to listen to Allan Muir, International Pompe Association and Pompe Support Network UK, talk about what he has learned at this year’s event, with Raymond Saich, International Pompe Association and Australian Pompe Association.

The webcast is also available at this link (courtesy of the Pompe Support Network).

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