By Sophie Muir | Chair of Trustees
In October, Timothy Syndrome Alliance (TSA) were thrilled to announce that we had been selected as one of 30 family-driven rare disease organisations to receive funding as part of the third cohort for the Chan Zuckerberg Initiative Rare As One Network. Following a competitive international application process TSA was awarded a five-year $800,000 USD grant to accelerate research and improve outcomes for patients living with rare diseases, specifically CACNA1C-related disorders, by strengthening communities, building capacity, and promoting collaboration.
“With the addition of international patient-led organizations, CZI’s Rare As One Network is exploring how to scale its model of supporting and elevating patient organizations to help address the global burden of rare diseases.Rare disease knows no borders. With up to 10,000 rare diseases affecting over 300 million people globally, our community response must also be global.Around the world, thousands of patient-led rare disease organizations do the hard work of improving the lives of people impacted by rare diseases and advancing medical research in their respective fields. But many of them face common barriers to progress, including limited organizational capacity and funding, small and scattered patient populations, academic silos, and insufficient data sharing and research infrastructure.Moving away from individual organizations attempting to overcome these challenges solely on a disease-by-disease basis, CZI’s Rare As One Network was founded to bring patient communities together to learn from one another, share resources, and collaborate to address common challenges. Grantees receive funding, support in organizational capacity building — such as deep dives in finance, operations and major donor fundraisings, as well as mentorship, science advising and training — along with access to a robust network of peers and the opportunity to participate in a forum discussion board, regular network calls and in-person convenings.”
Excerpts from the CZI Our Rare Disease Network Goes Global announcement blog, link below.
Our growing community is integral to the forward momentum in understanding CACNA1C. Their unique insights, perspectives and engagement in research are evident through our united front with our Scientific Advisory Board, both in research advocacy and stakeholder networking. We are tremendously grateful to the Chan Zuckerberg Initiative Rare As One Network for recognising TSA’s impact on our global community.
It is because of your support and generosity in recognising the importance of ensuring our registry's long-term sustainability and success that we are able to drive meaningful progress. Thanks to you, our growing global CACNA1C Community Registry is connecting families and transforming real-world healthcare data into valuable evidence. This registry serves as a pivotal tool in the development of our CZI-funded interactive portal, which will make aggregated CACNA1C disease data accessible to the broader global scientific and clinical community. Most importantly, it allows us to continue working toward our vision of a world where shared knowledge and understanding lead to a cure for everyone with a CACNA1C genetic variant.
Thank you for being with us on this exciting journey.
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