Good Vibrations? Posted On: 10/03/2024 Reports of experiences with vibration technology by Pompe patient There has been some interest lately about vibration technology in relation to Pompe. Kevin Annesley, PSN board member, has written about his experiences. Read More...
Aro Biotherapeutics gets FDA orphan drug designation for treatment of Pompe Disease Posted On: 11/08/2022 Aro Biotherapeutics gets FDA orphan drug designation for treatment of Pompe Disease Read More...
What the development of Myozyme meant for patients, their families, and their community Posted On: 07/04/2022 The story of the UK Pompe community’s involvement in the development of enzyme replacement therapy for Pompe disease is the subject of an academic publication. Read More...
Novel Oral Therapy for Pompe disease Posted On: 31/03/2021 A new player, MAZE Therapeutics, has joined the growing number of companies with an interest in Pompe disease. This time they are targeting Muscle Glycogen Synthase, the enzyme that causes a very rare muscle form of Glycogen Storage Type 0. Read More...
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 Read More...
UK Studies for Pompe disease Posted On: 08/05/2020 *Research Update - 17/09/20 There are a growing number of clinical studies for next-generation Enzyme Replacement Therapies, Gene Therapies. These are usually reported on US, EU and WHO websites, but we have selected those with UK study centres and will update our list to keep it relevant. Read More...
Notes from SSIEM 2019, Rotterdam, 3-5 September 2019 Posted On: 08/05/2020 Allan Muir, Pompe UK, Allan Muir attended the 2019 symposium of the Society for Study of Inborn Errors of Metabolism (SSIEM) on behalf of the UK LSD Collaborative. Read More...
