A doctor without borders

UF professor helping to improve care for kids with GSD in China

By Michelle Champalanne

In December, David A. Weinstein, M.D., M.M.Sc., spent two weeks in China meeting with health professionals, giving lectures and seeing patients, all with one goal in mind — improving care for children with glycogen storage disease in the world’s largest country.

Because glycogen storage disease is so rare — affecting just one in 100,000 people in the United States — there are few centers that specialize in the treatment of the disease. UF’s Glycogen Storage Disease Program, which Weinstein directs, is the largest of its kind in the world. The program cares for almost 500 patients from 49 states and 39 countries.

During a trip to China last year, Weinstein had noticed the children there were not receiving the proper treatment. He worked to strengthen and improve the care available locally to raise survival rates. In December, Weinstein visited Hong Kong, Guangzhou and Shanghai to meet with doctors and educate and train them on the proper medical management of children with glycogen storage disease.

“It’s shocking at this point at how poorly the children are doing,” he said. “But I’m very optimistic that it’s going to change the outcome for the children.”

The disease is so rare that most parents in China didn’t realize glycogen storage disease was treatable, he said. Now, he is working with doctors to ensure children no longer suffer from this condition. His collaboration with Chinese physicians is modeled after other GSD centers Weinstein has established in Brazil, Spain and the Faroe Islands. He is also working to create treatment centers in India.

The new centers have proved successful in improving not only the quality of treatment, but also the chances of survival for patients.

“I’m very optimistic we’re going to take a disease that was a fatal condition and now have healthy children,” he said.