The IPA has authored an article titled “The role of patient advocacy organizations in shaping medical research: the Pompe model,” and we are excited to announce that it has been accepted for publication.

The article focuses on the Pompe Model, a term describing the long-standing relationship between the patient community, the medical/scientific community, and industry. Over the last 20 years, we have seen that as a united community of patients, family, friends, physicians, scientists, and industry, working together, we have an undeniable strength and we have made, and will continue to make, great strides in learning more about Pompe Disease and in working towards treatments and disease management.

The article is published in the Annals of Translational Medicine, an international, peer-reviewed  journal, and can be found here.


The Pompe model is the term used by the Pompe community to describe the relationship that exists between the patient community, the medical/scientific community, and industry. The development of the Pompe model represented a new paradigm for the involvement of patients in new treatments—and also for scientists and pharmaceutical companies. It saw patients developing a sense of agency, of involvement in the process of treatment development rather than powerless recipients or (if lucky) occasional spectators. At the same time, as described below, it benefited the other partners in the process with the result that the different components of the model added up to more than the sum of their parts. However, in order for this to happen, each part had to undergo a transformation in mindset. The development of enzyme replacement therapy (ERT) for Pompe disease represented a unique set of circumstances and individuals that helped to bring about this change and, in doing so, created a model that has had far wider applications.

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