UPDATE: We currently have to raise the funds necessary to manufacture the treatment and perform a toxicology study that will help determine the dosage to be used in the human trials. Contributions go toward paying that bill!

As soon as we pay that bill, we will begin a ~2 year countdown until our George could receive the drug that would halt progression of his disease! We will need to raise a lot more for the following stages, but our immediate goal feels very much within reach.

Our story:

In February 2025, we were devastated to learn our four year old George was born with a neurodegenerative disease called CMT4C. It is gradually deteriorating his mobility, use of his hands, hearing, chewing, and breathing. We were told all we could do was pursue supportive care and that most children with CMT4C lose their ability to walk independently in their teens.

Out of that darkness, we were encouraged to learn of a decade-long pre-clinical study (funded by the NIH, the CMTA, and the MDA) that demonstrated the efficacy of an intrathecally delivered AAV-mediated gene therapy. The data showed treatment could not only stop the progression of George's condition, but even reverse some of the degeneration if administered early enough.

Now, funding is needed to advance the gene therapy towards FDA approval and human clinical trials. Project Foresee is accelerating this development.

Your contributions will fund drug manufacturing and formal preclinical toxicology studies, both critical components of the IND (Investigational New Drug) submission process with the FDA.

Every contribution brings us closer to stopping the progression of this cruel disease for George and the thousands of other Americans with CMT4C, many of whom are young children. The sooner George has access to this drug, the more function will be preserved for the rest of his life.