Research

On this page we will publish news of interesting research as we become aware of it. That may be from press releases, published papers or our own reports from attending meetings and conferences.

Pregnancy Outcomes in Late Onset Pompe Disease

Posted On: 09/06/2021

There is limited data on pregnancy outcomes in Pompe Disease (PD) resulting from deficiency of the lysosomal enzyme acid alpha-glucosidase.

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Could there be more than 2,500 people living with Pompe disease in the UK?

Posted On: 19/05/2021

A new study has been published in the journal Molecular Genetics and Metabolism suggesting that the prevalence of Pompe disease could be much higher than previously thought.

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Would you like to help research into Pompe disease?

Posted On: 16/02/2021

If you have Late Onset Pompe disease and are not already taking part in a clinical trial for Pompe, a research team at a Newcastle study would be interested to take a blood sample from you to help them understand how antibodies in patients may affect a particular gene therapy.

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MHRA deems Sanofi’s avalglucosidase alfa ‘promising’ for Pompe disease

Posted On: 01/10/2020

The PharmaTimes online has reported that the MHRA have issued Sanofi a Promising Innovative Medicine designation in the treatment of Pompe disease.

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New Treatment Developments for Pompe Disease

Posted On: 30/09/2020

A Letter from Allan Muir, Chair, Pompe Support Network

I’ve been wanting to write this article for some time but was slightly daunted by the number of potential treatments currently under study; some paused due to Covid-19, and a few reopening their clinical trial sites. So, it was a wonderful surprise to receive notification of a paper published recent

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