“Living with Late-Onset Pompe Disease in the UK”

“Living with Late-Onset Pompe Disease in the UK”

: interim results characterising the patient journey and burden on physical, emotional and social quality of life

Posted On: 24/09/2021

Allan Muir, Chair of the Pompe Support Network, presented the Poster “Living with Late-Onset Pompe Disease in the UK” at the World Muscle Society on Thursday 23rd September 2021.

The poster, sponsored by Amicus Therapeutics, was produced from an analysis of interviews of 27 LODP individuals living in the UK. It collates the emotional, diagnostic and treatment journeys together into an original and fascinating form that highlights the emotional roller-coaster experienced by people within the Pompe community.

We would like to express our extreme gratitude to all those who took part in this project; the information will be invaluable in many situations; for example, in generating educational material for families, individuals, healthcare professionals, and medicine regulators.


Pompe Disease News has published news of this survey, showing how adults with late-onset Pompe disease in the U.K reported a long and distressing diagnostic journey, with most patients calling for reduced time to diagnosis, shorter wait for a specialist appointment, and earlier treatment start.

To read the article please click here:

Patients: Faster Diagnosis, Earlier Treatment, Better Education Needed