Programs

  • Clinical Assessments Research Study

    In 2024, the CACNA1A Foundation launched a 10-year Clinical Assessments Research Study to establish baseline measurements for clinical trials and advance treatment development. We funded multiple research projects, including novel gene therapy strategies, small molecule screening, and antibody validation, while also expanding our Research Network with new working groups. Additionally, our team published a key paper outlining a pathway to clinical trials for CACNA1A-related epilepsies.
    Geographies
    Not indicated
    Dates
    Jan 1, 2024 – Dec 31, 2024
    Source
    990
    No causes provided
    No populations provided
    –
    $247.5K

  • Research Roundtable and Community Conference

    On July 18, 2024, the CACNA1A Foundation hosted its third annual Research Roundtable, bringing together 50 researchers, clinicians, industry representatives, and NINDS grant managers to advance clinical trials for CACNA1A-related disorders. We also held our second hybrid Creating Connections Community Conference, uniting over 200 participants, including families and experts, to share research updates and support. This event remains a vital space for connection, education, and empowerment in the rare disease community.
    Geographies
    Not indicated
    Dates
    Jan 1, 2024 – Dec 31, 2024
    Source
    990
    No causes provided
    No populations provided
    –
    $159.6K

  • Awareness and Advocacy Initiatives

    The CACNA1A Foundation raised awareness by participating in national and international events, including Global Genes, the American Epilepsy Society, and Rare Disease Day at the Broad Institute. We hosted 22 virtual meetups for families and launched the Cure Club to boost research participation through personalized outreach. Additionally, we advocated for a unique ICD-10 code for CACNA1A-related neurodevelopmental disorders at the ICD-10 Coordination and Maintenance Committee Meeting.
    Geographies
    Not indicated
    Dates
    Jan 1, 2024 – Dec 31, 2024
    Source
    990
    No causes provided
    No populations provided
    –
    $34.6K