Nexviazyme is under review by Medsafe NZ

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...

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2022 RARE Patient Advocacy Summit

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,...

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WORLD Symposium 2023

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...

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Last blog from the USA

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...

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Counting down to home

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...

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