What will a cure for Fragile X look like? It’s a question we all ask at some point. Will it be a pill taken every day for life? Will it be combinations of medicines, tailored to each individual? Will it be a protein replacement injection once a week? Will it be a one-time gene therapy infusion that fixes every cell in the body at the same instant?
The answer is probably all of the above. The cure for Fragile X isn’t a pipe dream. It’s a process, and we are right in the thick of it.
Engineering a Cure for Fragile X
What FRAXA does goes far beyond what most people think of as “research.” We are running a massive engineering project. We are constantly troubleshooting this complex project and making adjustments. Although this is a daunting task, we have validated dozens of potential therapeutic strategies which are now ready for clinical trials. Indeed, that’s where the backlog is today: we have far more treatment candidates than we have clinical trial capacity.
What FRAXA does is unique and essential to engineering a cure. These FRAXA-funded investigators and many, many others are hard at work at this very moment because of donors like you. Let’s keep them working. Let’s add more teams so that we have the best possible chance of bringing hope to families and friends affected by Fragile X.
Dr. Tranfaglia is Medical Director and Chief Scientific Officer of FRAXA Research Foundation, coordinating the Foundation’s research strategy and working with university and industry scientists to develop new therapeutic agents for Fragile X. He has a BA in Biology from Harvard University and an MD from the University of North Carolina at Chapel Hill. His son Andy has Fragile X syndrome.