As with 2020, 2021 has been a difficult year for all of us, as patient representatives, patients, caregivers and personally with our friends and family. We have not been able to meet up, spent time alone in our homes and had disruptions to treatments, clinical care, and our jobs; however we have also achieved so much for our community, and we should take few minutes to remember that.
2021 saw us strengthen our membership with two new members from Portugal and Thailand, making our membership 57 strong. Despite no face-to-face meetings the IGA was present and participated in many international meetings, patient advisory boards, educational workshops, and webinars to ensure that the voice of the Gaucher patient is strong and present so that the needs of our community are heard and actioned to the best of our ability.
We are particularly excited to be working alongside the IWGGD on various initiatives including GD international guidelines, home therapy, newborn screening and a series of workshops on ethical dilemmas in Gaucher disease.
With a diverse and changing world we have been successful in increasing our reach and global footprint to support our community with new regional managers, all volunteers, plus volunteers joining our project groups, such as home therapy, Go with Gaucher, translations, older generation, and co-morbidities.
We have seen a new treatment in clinical trial for type 2 Gaucher disease. With such a huge unmet need this trial is very important and we will watch and wait for the data to be available. We have also seen further recruitment for a GD1 gene therapy trial and anticipate new trials in GD1 and GD3 for 2022 with serial modalities.
Finally, we are putting the final touches to GARDIAN, our patient owned, patient led nGD global registry which we will open in January 2022 and can’t wait to accept patient zero into the platform.
2022 is now upon us and we are excited to be hosting a face-to-face members meeting in May in Leiden, Netherlands. We do hope that all our members will be able to send their representative and hope to invite some new fledging organisations that are considering joining us.
As always, we have a demanding annual programme and will be seeking financial and manpower resources to support it; we will share this with you in the New Year.
I hope you were able to take time for yourself over the holidays and remember that in 2021 you made a difference in the world. 😊
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