What is MPS I?

Mucopolysaccharidos1 (MPS I) is a rare genetic disease affecting one child out of every 100,000 births. MPS I patients lack a critical enzyme the body needs to break down certain proteins. Unchecked, the protein build up progressively damages the respiratory system, organs, joints, bones and central nervous system.

We're helping win the race.

Find the research. Raise the dollars. Provide hope.

When Jeanne and Mark Dant were told their son Ryan had Mucopolysaccharidos 1 (MPS I) at age 3, it was a double dose of the worst news parents could ever expect to hear. The bad news was their son now had a life expectancy of 10 years and, during those years, they could expect to see their son's health gradually decline while pain gradually increased. The worse news was not only was there no cure, there was no one even looking for one because their son's disease was so rare, there could be no profitability in a cure even if it could be found. Their only options: accept the inevitable or fight the indomitable. They chose the latter and devised a two-pronged battle plan.

Knowing whatever treatment or research that might save Ryan's life would require funding, they instantly set forth fundraising. Their first effprt, a bake sale, netted $342 and the Ryan Foundation was born. With the help of friends, neighbors and local businesses, bake sales soon gave way to golf tournaments, fishing tournaments and raffles. With awareness of their mission growing through local and national media attention, the foundation received $200,000.00 from an individual donor.

Enter the second part of the plan: find any research capable of leading to even a treatment for MPS I. Tenacity, persistence and tireless medical sleuthing led the Dants to the UCLA laboratory of Dr. Emil Kakkis. Dr. Kakkis had the beginnings of a treatment but his research was stalled due to a lack of funding. With the help of the Ryan Foundation, Dr. Kakkis and his small team of dedicated researchers continued their steady, gradual movement toward the treatment those suffering from MPS 1 were so desperately seeking.

By late 1997, Dr. Kakkis’ work caught the attention of newly forming BioMarin Pharmaceutical. Soon, a partnership was reached which allowed Kakkis to finish his research and get a promising new drug therapy into clinical trials. Ryan and nine other brave children from around the US, each suffering deeply from the effects of MPS I, volunteered to travel to the Harbor UCLA Medical Center in Torrance, California, just a few hundred yards away from Dr. Kakkis’ lab, to start taking weekly IV infusions of Dr. Kakkis’ new drug. The resulting new drug, Aldurazyme, almost immediately started reversing the progression of MPS I symptoms for Ryan and other trial participants.

In 2000, the FDA called for a second trial, and 50 more incredibly brave children from all over North America and Europe repeated what the original ten had gone thru in the previous two years. In February 2003, the Dants and several other families from both trials, traveled to Washington DC and spoke at the FDA Hearing to approve the use of Aldurazyme to treat MPS I. The panel voted unanimously to approve. Soon, children all over the world with MPS I began to improve with Aldurazyme therapy.

The race to beat the clock for Ryan was temporarily won, but Aldurazyme is not a cure. The damage caused to the central nervous system, heart valves, bones and ligaments in MPS I patients still needs to be addressed. That's why more science, more funding and more hope are needed.

Along the journey, the Ryan Foundation became aware of many more children facing other orphan diseases such as MPS I. Today, the Ryan Foundation continues to raise awareness and funding for orphan disease research affecting children and parents around the world. Much of the science already funded by the Ryan Foundation has now translated to other diseases like Hunter Syndrome, Morquio Syndrome, Maroteaux-Lamy Syndrome, to name just a few. The Foundation also works to help streamline the processes and legislation that slow the development and introduction of life-saving drugs targeting orphan diseases.

The Ryan Foundation has no paid employees, owns no property, and ensures that 100% of all net proceeds are forwarded to scientists working to treat and eventually cure orphan diseases.

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Ryan Dant and Dr. Emil Kakkis