We launched the first patient data collection program for v-ATPase-related disorders in partnership with Rare-X on March 19th, 2024. This milestone is the first step stone in our journey towards understanding and treating v-ATPase-related disorders.
The v-ATPase Alliance patient Data Collection Program (DCP) is a foundational study to create a robust open-access data set to speed scientific breakthroughs and life-changing treatments for our children. We aim to gain important information that may help in understanding how these disorders manifest, who is "at risk" of developing certain epilepsy and neurodevelopmental symptoms, who is not, and why, and we will follow people over time to learn how the disease progresses during its natural course. That information may lead to insights and tools that can help better diagnose, treat, and even cure this disease.
But we can't do it alone.
We need your enrollment and participation to gather important data from as many individuals as possible - remember we are a small population and therefore each participant has a huge impact on the robustness of the study. This is a tremendous task that our team is committed to supporting moving forward. The v-ATPase DCP study will set the foundation to establish Clinical Outcomes Assessments (COAs) and Clinical Endpoints. These consist of measurements or observations that reflect how a patient feels, functions, or survives, and outcomes that can be used to evaluate the effect of an intervention on participants in a clinical trial, respectively.
The v-ATPase DCP is pivotal for our long-term success, enabling us to:
Together we have the ability to build a vast and evolving engine of discovery that will funnel a wealth of insights, practical tools, and data directly into the hands of researchers and drug developers worldwide. The v-ATPase DCP holds the promise of reshaping the trajectory of v-ATPase research, guide clinical decision-making, and contribute to a healthier future for our children.
Sign up today and complete all the questionnaires from the comfort of your home.
Help us cure v-ATPase disorders.
Want to know more about the v-ATPase DCP?
Watch our launching Webinar!
Follow our Page by Page Guide with notes to help you guide through the setup and consent forms. PDF available here.
We're starting to get the first pieces of data being collected by our v-ATPase Data Collection Program on a rolling basis. Importantly, all this de-identified data is freely available to researchers around the world on an individualized level to help investigating and defining the condition's presentation and progression. If interested in studying this in detail, please reach out to us and we can help setting you up to access the data. This is a critical initiative to define this condition and support development of treatments.
*Preliminary Results, June 2024
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