Reagan is a happy and energetic 2-year-old girl who loves to dance, play and have tea parties. She makes everyone around her feel so joyful that it is hard to believe she has recently been diagnosed with a rare and fatal genetic disorder, MPS, otherwise known as Sanfilippo Syndrome.

Sanfilippo Syndrome results from defects in a lysosomal enzyme. Children with this disease are unable to appropriately break down sugar which causes carbohydrates to accumulate throughout the somatic and central nervous system. In other words, these children who appear normal at birth will later show severely delayed neurological development and become unable to talk, walk and even feed themselves. Children with Sanfilippo Syndrome oftentimes do not live past 20-years old.

Even though there is no cure or treatment for Sanfilippo Syndrome, there is hope. Some of the leading research on this fatal disease is taking place at Nationwide Children’s Hospital, with the expectation of a possible gene therapy treatment going to human clinical trials soon.

The Research Institute at Nationwide Children’s Hospital is one of the fastest growing pediatric research centers in the United States and is ranked in the top 10 for National Institutes of Health funding among free-standing children’s hospitals. The Institute is organized into 12 Centers of Emphasis that allow traditional academic boundaries to be crossed and merged to facilitate interdisciplinary research. Among these centers is the Center for Gene Therapy where the research is taking place on Sanfilippo Syndrome. “

After hearing such devastating news only a few weeks ago, we are now filled with hope because of the wonderful doctors and research going on at Nationwide Children’s Hospital. We are in a race against time to save our daughter, and I know the doctors and staff at Nationwide Children’s are in the race with us. It gave us the feeling that we are not in this alone, and that the people at Nationwide Children’s truly care for our daughter like one of their own,” says Reagan’s mother, Karin.

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• Name: Reagan M.Reagan McGee
• Condition(s): Sanfillipo Syndrome
• Age at Treatment: 2

Reagan is a happy and energetic 2-year-old girl who loves to dance, play and have tea parties. She makes everyone around her feel so joyful that it is hard to believe she has recently been diagnosed with a rare and fatal genetic disorder, MPS, otherwise known as Sanfilippo Syndrome.

Sanfilippo Syndrome results from defects in a lysosomal enzyme. Children with this disease are unable to appropriately break down sugar which causes carbohydrates to accumulate throughout the somatic and central nervous system. In other words, these children who appear normal at birth will later show severely delayed neurological development and become unable to talk, walk and even feed themselves. Children with Sanfilippo Syndrome oftentimes do not live past 20-years old.

Even though there is no cure or treatment for Sanfilippo Syndrome, there is hope. Some of the leading research on this fatal disease is taking place at Nationwide Children’s Hospital, with the expectation of a possible gene therapy treatment going to human clinical trials soon.

The Research Institute at Nationwide Children’s Hospital is one of the fastest growing pediatric research centers in the United States and is ranked in the top 10 for National Institutes of Health funding among free-standing children’s hospitals. The Institute is organized into 12 Centers of Emphasis that allow traditional academic boundaries to be crossed and merged to facilitate interdisciplinary research. Among these centers is the Center for Gene Therapy where the research is taking place on Sanfilippo Syndrome. “

After hearing such devastating news only a few weeks ago, we are now filled with hope because of the wonderful doctors and research going on at Nationwide Children’s Hospital. We are in a race against time to save our daughter, and I know the doctors and staff at Nationwide Children’s are in the race with us. It gave us the feeling that we are not in this alone, and that the people at Nationwide Children’s truly care for our daughter like one of their own,” says Reagan’s mother, Karin.