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Molecular Medicine And Diabetes

Lilly Jaffe a six-year-old girl diagnosed with type 1 Diabetes was admitted to the Clinical Research Center at the University of Chicago Medical Center on August 14. // Four days later the North Shore suburban girl was well on her way to manufacturing insulin in her own body and ready to spend a few days at the beach before she started school.

As she continued to do well, her insulin was reduced day by day. The following Wednesday, August 23, with her doctors' direction, Lilly's mother disconnected her insulin pump--the lifeline and security blanket she had relied on for years--for the last time.

"She is so proud," said her mother, Laurie Jaffe, "so happy, so excited. She's just thrilled, for the first time to be like her friends and her brother and sister."

"It is a miracle," she added. "We are humbled by the scientific brilliance and by the amazing grace of God that brought this cure to Lilly."

"It was awesome," said her doctor, diabetes specialist Louis Philipson, professor of medicine at the University of Chicago. "It was cool," he added, with uncharacteristic abandon, "way cool."

It was also lucky. Lilly suffered from an unusual form of diabetes caused by a genetic mutation rather than the errant immune system responsible for type 1 diabetes. Such "monogenetic" forms of diabetes in children are just being recognized and studied.

Lilly is only the fourth such case treated in the United States and one of less than 100 in the world who have been successfully treated this way.

Researchers suspect there are about 2,000 people in the United States with neonatal diabetes who could benefit from the same treatment if precisely diagnosed and treated relatively early in life.

The take-home message, Philipson said, is that "anyone who has what appears to be type 1 diabetes with onset before the age of six months should be tested for this condition."

Conventional type
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