Funding

The funds received through Ryan's Quest are directed, in full, to Duchenne muscular dystrophy research. Ryan's Quest bases all decisions regarding the awarding of funds to research projects, by speaking with lead doctors, researchers and scientists concerning current and promising advances being made in Duchenne research. We also collaborate with other non-profit organizations which have similar goals to that of Ryan's Quest. These organizations also have scientific advisors who specialize in the field of DMD to determine the validity and potential of a given project.

In addition, Ryan's Quest has gained the knowledge and expertise of our very own medical advisor, Dr. Joseph Perno. Dr. Perno completed his PhD in Physical Chemistry from Princeton University, Princeton NJ where he was a Garden State Scholar and received his MD from Duke University School of Medicine in 3 years, where he held the Joseph Barham Scholarship. He is a board certified Diagnostic Radiologist and currently Clinical Assistant Professor of Radiology at the University of Pennsylvania. Dr. Perno's ability to converse with other medical experts and researchers in the field of DMD is a great asset to this organization.

CombinatoRx is a unique pharmaceutical company based in Cambridge, MA. The company focuses on developing new medicines from combinations of existing FDA-approved drugs that can work together to achieve a desired effect. CombinatoRx has a team of six scientists devoted to developing a treatment for DMD. They are looking at millions of combinations of drugs that have been approved for other uses to see if any of the compounds can work in tandem to slow or stop the relentless progression of DMD. This research is funded by several DMD foundations, including:
• Charley's Fund (South Egremont, MA)
• The Nash Avery Foundation (Minneapolis, MN)
• The GM Trust (London, UK)

Brown University -- Justin Fallon, PhD
Dr. Fallon has developed a drug called Biglycan, which stimulates the cells affected by DMD, forcing them to continue producing utrophin which is an important protein people normally lose around the age of four or five.
Utrophin is a compensatory protein that can act as a substitute for dystrophin, the missing protein in DMD boys. Dr. Fallon has discovered that biglycan can upregulate utrophin expression in a muscular dystrophy mouse model. He has observed other beneficial effects of biglycan, including reduction of muscle fiber cell death. Dr. Fallon is now scaling up the production of biglycan in preparation for preclinical safety studies.