Breakthrough Treatment for Rare Liver Disorder

When Julie’s son, Andy, was 4 months old, she noticed he suddenly became lethargic and unresponsive. After being rushed to the hospital, Andy fell into a coma, from which he quickly recovered, though Julie was extremely concerned about what could be causing his sudden illness.

“Every time I put him down at night, I was terrified he wouldn’t wake up the next day,” she says.

For years, Andy was in and out of the hospital and was constantly monitored by doctors. At age 4, Andy finally received a diagnosis: Glutaric Aciduria Type II, an extremely rare, inherited liver disorder that interferes with the body’s ability to break down proteins and fats to produce energy, resulting in a buildup of fatty acids and other harmful substances in the body. The condition occurs in approximately 1 to 9 out of every 1 million births.

Julie says her son has been hospitalized more than 500 times throughout his life. Although a cornstarch treatment helped alleviate his symptoms for several years, the regimen lost its effectiveness when Andy was 10 years old, and his liver became less functional as the years wore on. Eventually, Andy’s liver started to fail, and his doctors gave him just 6 months to live. The 29-year-old didn’t have time to wait for a cadaver organ transplant, so his doctors asked Julie if she would agree to donate a portion of her liver to her son.

“There was no question,” Julie says. “Of course I would do this to save my son’s life.”

Live donor liver transplantation is a relatively new procedure, which only a handful of centers in the United States perform. Andy is the first glutaric aciduria patient to receive this form of treatment. As the longest living person with the condition, he will continue to be monitored to determine the long-term effectiveness of the transplant. 

Onstage with The Doctors just a few weeks after surgery, Andy says he hopes his story can help save the lives of individuals with his condition in the future.

 

To learn more about becoming an organ donor, visit organdonor.gov.

 

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