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...
Experiences with Electrical Muscle Stimulation Posted On: 31/01/2024 A Pompe Support Network pilot study of the Mollii Suit The Pompe community has many individuals who have worked with physiotherapists and undergone their own experimentation to discover exercises or other manipulation strategies that may improve muscle function, strength, and endurance. Pompe Support Network organised assessment days of the Mollii Suit 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...
Ketones and Pompe Posted On: 05/08/2022 Could this new study provide the benefits of a Ketogenic diet in Pompe Disease? Read More...
Muscle MRI in Pompe disease Posted On: 22/07/2022 - Would you like to help research into Pompe disease? A research group led by Prof. Jordi Díaz-Manera at the John Walton Muscular Dystrophy Research Centre in Newcastle is currently recruiting UK Pompe patients for an important study of skeletal muscles. Read More...
Small fibre involvement is independent from clinical pain in late-onset Pompe disease Posted On: 21/06/2022 A recent study has been carried out with the aim to analyse the pain characteristics and its relation to a small nerve fibre involvement in Late Onset Pompe patients. Read More...
Experience the benefits of a Mollii Suit at our Coventry assessment day Posted On: 26/05/2022 Come and view the Mollii Suit in action with our Pompe get-together! 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...
UK Research study for adults living with Pompe disease Posted On: 09/02/2022 Barriers and facilitators to Physical Activity and exercise in Late Onset Pompe Disease (LOPD) A qualitative study by Nicola Condon exploring patient perspectives and experiences. Read More...
Pompe quality of life: Future Research Priorities Posted On: 08/07/2021 To help Pompe Support Network understand your research priorities we've created a simple form which we'd appreciate your response. Read More...
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. Read More...
Invitation to UK patients to participate in research into patients' and families' experience of living with Pompe disease Posted On: 03/06/2021 An invitation for people in the UK living with Pompe disease to help with research which will explore the effects of Pompe disease on families. Read More...
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. Read More...
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. Read More...
Spark Therapeutics Announces First Participant for LOPD Posted On: 01/02/2021 Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease. Read More...
AVROBIO Announces New Positive Clinical Data and Preclinical Data, as Well as Expanded Leading Lysosomal Disorder Gene Therapy Pipeline Posted On: 18/11/2020 - AVR-RD-03 for Pompe disease: Preclinical data show normalization of substrate levels in multiple hard-to-reach organs 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...
