FRAXA is a truly remarkable organization with only one goal in their mission statement: to find a cure for the genetic disorder, Fragile X. Presented with a zero public recognition factor on day one, the dedicated founders and staff at FRAXA have moved the Fragile X
Mission: FRAXA was founded in 1994 by three parents of children with Fragile X. Our mission is to accelerate research aimed at finding effective treatments and a cure for Fragile X and related disorders, by directly funding grants and fellowships at top universities around the world.
Results: We have funded $23 Million in targeted research and are on the verge of bringing a treatment for Fragile X - and perhaps also autism - to patients.
Target demographics: find effective treatments and ultimately a cure for all children and adults who have Fragile X syndrome
Direct beneficiaries per year: 20 research teams to find new treatments for Fragile X; half of them have now gone on to receive large grants from the National Institutes of Health.
Geographic areas served: the U.S., Canada, Europe, South America, and Australia
Programs: 1) Research grants and fellowships to fund research on Fragile X Syndrome, autism, and related disorders at universities around the world.
2) The FRAXA drug validation initiative (FRAXA-DVI), where we can provide preclinical testing of potential new medications for Fragile X; we have provided this service to a dozen pharmaceutical companies and now several of them are conducting clinical trials.
FRAXA is a truly remarkable organization with only one goal in their mission statement: to find a cure for the genetic disorder, Fragile X. Presented with a zero public recognition factor on day one, the dedicated founders and staff at FRAXA have moved the Fragile X conversation forward. America can boast a National Fragile X Recognition Day due to this group. As the parent of a Fragile X young man, I know that my contribution to FRAXA makes a difference. Why? Because contributions fund research grants, not administrative costs. Medical, genetic, and pharmaceutical researchers around the world are working toward a cure. My son will be 26 years old in 2 days. Go FRAXA! You are doing the impossible, and everyday we thank you for the cure that is coming.
I have interacted with FRAXA since 2006, acting as a researcher working in development of new medicines for Fragile X Syndrome. What attracted me to FRAXA in the first instance, and continues to be true today, is the role FRAXA has played in putting Fragile X Syndrome on the radar of the pharmaceutical industry. I can remember the days when there were no clinical stage investigations of novel potential therapeutic approaches to Fragile X Syndrome. Now there is an industry wide pipeline of programs in development across many companies. I think it is a simple truth that without FRAXA this would not be the case. The initiatives FRAXA has in place have, and continues to be, crucial to this critical mass of effort to find new options for people with lives touched by Fragile X Syndrome.