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The Scripps Research Institute Season's Greetings
 
  Dear Friend of Scripps Research,

When most people think of superheroes they envision larger than life characters cloaked in masks and capes. But to the families of children with Phenylketonuria (PKU) -- a rare and debilitating metabolic disorder -- their hero has salt & pepper hair and wears a lab coat.

His name is Ray Stevens and he'll humbly tell you that he's just a researcher at Scripps. But that hardly sums up the man who is leading the way to improving the lives of thousands of children.

 
  This holiday season, continue to help all the heroes at Scripps Research provide hope.

Because of friends like you, a second PKU drug developed by Ray Stevens will go into clinical trials next year… eventually giving doctors more treatment options.

It's just one more piece of hope that you helped Ray give these families.


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It all began in the 1990's when Ray was conducting basic metabolic research at Scripps Research and discovered mutations in the gene that is responsible for PKU. At that point, the only way to control PKU was a strict, low-protein diet. Because PKU patients are unable to process a part of a protein called phenylalanine (Phe), which is found in almost all foods, their lives become drastically limited. However, if Phe levels get too high, it can cause severe mental retardation.

But Ray's discovery finally offered a promising opportunity to find a treatment and maybe even a cure. The only problem was funding. Because PKU affects less than 200,000 people a year, it's classified as an "orphan" disease. And these diseases typically don't get "adopted" by the pharmaceutical industry because they provide little financial incentive.

For families dealing with PKU, and all of us at The Scripps Research Institute, this was unacceptable. But without funding, the opportunity to develop a new drug seemed unlikely.

ResearcherAnd then fate brought the scientist and these families together. It started with an invitation to discuss his PKU research at a fundraiser. Soon, Ray's determination to find a treatment turned into a passion. When he wasn't in his Scripps Research lab, he was participating in walk-a-thons, PKU picnics, and an array of fundraising events… all to raise money for his life-changing research.

The hope that Ray's research offered these families fueled a truly amazing outcome. From that initial fundraising effort, Scripps Research was able to collect enough data to apply for, and receive, a 1.2 million dollar grant from the National Institutes of Health to develop a drug for PKU! And it's most fitting that Kuvan -- the first-ever drug for PKU -- will finally be available to patients this month.

It's an incredibly wonderful holiday miracle for thousands of children.

While many call Ray Stevens a hero, it's the generosity of donors like you that fuels the passion of our dedicated scientists -- and helped Scripps Research develop Kuvan. So this holiday season -- as thousands of kids benefit from a brand new drug -- we ask that you make a special renewed gift. Your holiday donation will help ALL the heroes at Scripps continue their pioneering, life-saving research.

From all of us at The Scripps Research Institute, have a happy and healthy holiday season!

Sincerely,

Wendy Scott Keeney
Vice President of Philanthropy

 
 
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