Nexviazyme is a 2nd generation Enzyme Replacement Therapy (ERT) for Pompe disease. Genzyme Corporation has put an application in to Medsafe NZ for assessment and approval. Then we need to cross our fingers that PHARMAC will approve it for funding! Nexviazyme status at Medsafe Information on...
Pharmac’s year in review leaves a lot to be desired
Gene therapy was the focus of half of the best-read stories this year by Lindsey Shapiro, PhD | December 29, 2022
In Touch Magazine onlineThe latest edition of In Touch magazine is...
Rare Life. Bonded Together September 12-14, 2022 San Diego, CA The first conference attended since WORLD in March 2020! It was great to be able to get out amongst people again. It was a full schedule with many topics discussed. I was inspired by all of those who strive to keep showing up,...
If you’re heading to the WORLD Symposium in Orlando in 2023, click the link below for the latest updates WORLD Symposium 2023 WORLDSymposium 2023 in Orlando will be the place to connect, collaborate, communicate and continue to make global progress in lysosomal disease research. The LIVE...
Allyson Lock New Zealand Pompe Network Allyson, President of NZPN, opened the conference and thanked everyone for coming. She introduced the committee members and the process that had been gone through to make it all happen. Attendees included 8 of 11 Kiwi patients and their families,...
Masterton, New Zealand – (February 25, 2017) The New Zealand Pompe Network is ecstatic to announce that Sanofi Genzyme has offered Pompe disease patients in New Zealand access to their International Compassionate Access Program (ICAP) for the drug Myozyme®. A huge THANK YOU to Sanofi Genzyme for...
This is my last blog from this side of the Equator. Update on our fundraising for Benjamin Revell; We raised $520 for Benjamin and Malia’s daughter. And, it is with great joy I can say that “Luisa Rose Revell” was born this week 🙂 Congratulations to the new parents. Exciting times! Sophia has...
Fundraising for Benjamin Revell I’ve put an auction up on my Face Book page for a service to each of my miniature stallions. Another 3 friends have also offered the same. Isn’t it wonderful how people come together at a time of need? We hear so many negatives in the news and various other...