Pompe Support Network

@CentenaryInst Centenary Institute receive funding for first therapy targeted at Pompe disease. Having been awarded $500,000 from CUREator, the researchers intend to first demonstrate the utility of their new technology for gene therapy in Pompe disease pompe.uk/pompe-news/cen…

Short UK LOPD exercise survey - aimed at the LOPD part of the UK community. We are collaborating with Stephen Dando, a specialist physiotherapist for Pompe at the National Hospital, London, to determine what exercises Pompe individuals are doing: pompe.uk/pompe-news/sho…

@amicusrx1 Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the United Kingdom pompe.uk/pompe-news/ami…

Got your holiday travel insurance yet? Travelling whilst living with a disability can be difficult and finding travel insurance can add to the frustration. We've compiled a list of insurers to help you to tick off your holiday to-do list. pompe.uk/support-and-ad…

The book we have been working on for the last 12 months, “This Little Thing I Do”, has now been printed and a copy sent to each UK subscriber. If you haven't yet received yours or would like to receive a copy please click the link to find out more: pompe.uk/pompe-news/thi…

@ISNSNeonatal Today, June 28th 2023, we celebrate the 3rd International Neonatal Screening Day (INSD2023) Neonatal screening saves lives. The INSD raises worldwide awareness about the importance of neonatal screening to speed-up diagnosis and treatment: neonatalscreeningday.org

We now have a @JustGiving page! You can now fundraise or simply donate to Pompe Support Network via JustGiving. Please follow the link below. pompe.uk/pompe-news/we-… #pompe #JustGiving #fundraise #donate #community

Patient Registries for Pompe Disease The International Pompe Association would like your assistance in conducting a brief (only a few questions) survey on the topic of Patient Registries for Pompe Disease. This should not take no more than 10 minutes: pompe.uk/pompe-news/pat…

The @NMCentre have released more details about the family support group summer gathering on Saturday 22nd July 2023. pompe.uk/pompe-news/fam…

Did you know you can help support us just by a click of a button? Going via @giveasyoulive before internet shopping means the charity will receive donations at no cost to you! Plus this week you can win vouchers and spot prizes! Click here to read more: giveasyoulive.com/join/pompe

Would a Virtual Reality Demonstration Day be useful to you? We are hoping to host a VR demo day but first we'd like to know if this would appeal to you? Would you be willing to try out the VR world to see the mental & physical health benefits? facebook.com/PompeSupportNe…

*Family support group gathering at the NeuroMuscular Centre, Winsford* The Neuromuscular Centre will be hosting a summer gathering on Saturday 22nd July 2023 which is open to families and charities affected by neuromuscular conditions. pompe.uk/pompe-news/fam…

Living with Pompe disease: ‘I could be still walking unaided if diagnosis hadn’t taken 17 years’ Zoe Baillie speaks to the @Independent of how she started having symptoms of Pompe in her late teens, but didn't receive her diagnosis until her mid-30s. pompe.uk/pompe-news/liv…

Maze Therapeutics Announces Exclusive Worldwide License Agreement with Sanofi for MZE001 @MazeInBiotech has announced an exclusive licensing agreement with Sanofi for MZE001, an oral substrate reduction therapy for the treatment of Pompe disease: pompe.uk/pompe-news/maz…

Pompe Support Network at the WORLD Symposium 2023 Watch our short film celebrating the WORLD Symposium 2023 held in Orlando, Florida. Pompe Support Network represented the UK Pompe community at the annual event. pompe.uk/pompe-news/pom…

@amicusrx1 Full approval of Amicus new ERT, Pombiliti plus Opfolda, expected this Summer. On Weds 26th April Amicus Therapeutics announced the EU Committee for Medicinal Products for Human Use (CHMP) have recommended approval of Opfolda for Pompe disease pompe.uk/pompe-news/ful…

Click the website link below to read how the Pompe Support Network marked International Pompe Day 2023 at the @NMCentre, Winsford with guest speaker Professor Mark Roberts (Consultant neurologist at the Salford Royal Hospital) pompe.uk/pompe-news/int… #Pompe #rare #community

Happy International Pompe Day to the global Pompe community! We are having a wonderful time at our Pompe spring meeting at the NeuroMuscular Centre catching up with faces old and new - full report to follow soon! We hope you too are marking the day in style. #Pompe #community

The International Pompe Association (IPA) is excited to announce it’s plans for International Pompe Day April 15th 2023. This year the focus is on raising awareness of the importance of movement and require input from members of the Pompe community. pompe.uk/pompe-news/int…

Pompe Webinars from AMDA Date: Tuesday, March 28, 2023 Time: 7pm BST Link to the Webinar description and registration page: amda-pompe.org/teleconference… The USA patient organisation AMDA hold very interesting webinars throughout the year. The next one concerns two important topics.