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 does not get the financial support many others do. We have got to put 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, Inc. and put together what we believe is a first-rate board with doctors and businesspeople for this exact purpose. Remarkable genetic and other scientific breakthroughs are around the corner and we need to get them to Morquio patients.
The money raised will be used to fund ongoing research and to jumpstart new research from the world’s leading Morquio and genetic disease scientists, some of whom we have already been funding for about two years and others with whom we’ve been in contact since Robert was diagnosed.
Current targets include, but are not limited to
- Continue to provide assistance to a leading research team to advance promising new AAV gene therapies that might not only help treat Morquio, but initial studies suggest could possibly reverse effects (as has occurred in mouse models)
- Work with organizations to create RFPs for financially incentivizing other medical advances that could make current treatments significantly more efficient, effective, and affordable or to discover and/or perfect new treatments
- Continue working with the CEO of a genetic research pharmaceutical company on ways to assist in getting promising genetic therapies from lab to clinical trial
- Recruit and incentivize leading scientists and researchers in other disease research fields to enter the Morquio treatment space