I'm raising £50000 to support families with a CAPRIN1 genetic mutation

Help Us Advance Research for CAPRIN1 Genetic Mutation
We are raising £50,000 to fund critical research and move towards clinical trials for CAPRIN1 genetic mutation, a rare and life altering condition that causes neurodevelopmental conditions including autism, ADHD, speech impairment, neurodegenerative conditions including epilepsy and seizures. This mutation has a profound impact on individuals and families.
CAPRIN1 is a key protein involved in mRNA translation and stability, playing a vital role in brain development, synaptic plasticity, learning, and memory. Mutations in the CAPRIN1 gene have been associated with rare neurological disorders. Notably, a recurring missense variant is linked to early-onset ataxia, affecting coordination and movement control, while loss-of-function variants result in a neurodevelopmental disorder, underscoring CAPRIN1’s critical role in brain function. Investigating CAPRIN1’s role in these conditions will enhance our understanding of its importance in maintaining normal brain function and the effects of its dysfunction. Further research into its mechanisms in both healthy and diseased states is essential for developing potential therapeutic strategies for these neurological disorders.
For many families, a CAPRIN1 diagnosis comes with more questions than answers—there is little research, no targeted treatments, and few resources for support. Parents, caregivers, and individuals living with this condition are often left feeling isolated, searching for hope in a medical landscape that has yet to catch up.
With your support, we can:
• Advance scientific research to better understand the role of CAPRIN1 in the body.
• Support progress towards clinical trials to explore potential therapies and interventions.
• Support families by providing resources, awareness, and advocacy to ensure no one feels alone in this journey.
Every donation, big or small, brings us closer to answers and breakthroughs that could change lives. Together, we can drive change and make a real difference. Thank you for being part of this mission!