How Your Dollars Will Make a Difference
We need funding and need it right now. There are thousands of rare diseases, and for a variety of reasons Morquio has traditionally not received the financial support many others do. We are putting Morquio Funding on the map, now, and it often takes communities like ours to do so with such rare diseases. We specifically undertook to start A Cure for Robert and put together what we believe is a first-rate board with doctors and business people for this exact purpose.
Where Does the Money Go?
The money raised going forward will be used to continue to fund ongoing research and to jumpstart new research for the world’s leading Morquio and genetic disease scientists. To date, we have pledged over $480,000 to five different accomplished research groups which, in total, are running 9 separate studies. Each study has a specific goal: to further research for a particular idea that could be the next (and better) treatment for Morquio. These researchers are investigating everything from repurposing currently approved FDA drugs to cutting-edge gene therapies.
Most of these studies would not happen without our support, and for those that have other funding sources our funding is of paramount importance to keeping them going. All dollars are subject to grant agreements that require periodic updates and protect our Non-Profit in the event of adverse events (such as deviations from proposed budgets, breaches by the recipients, or key principals leaving).