VietNamNet Bridge - They are pioneers in studying rare diseases and disseminating knowledge in the community. They do not work for money and have no interest in receiving honors.

Tran Minh Dien, deputy director of the National Hospital of Pediatrics

Many patients with rare diseases in Vietnam have to be treated with specialized imported drugs.

In the US, one out of every 1,500 newborns has a rare disease. In Europe, the ratio is 1/2000.

It is estimated that there are over 6,000 types of rare diseases, 80 percent of which are inherited, while the remaining are caused by infections (bacteria and viruses), immune disorders and environmental conditions. 

In Vietnam, 10 years ago, there was almost no concept of these diseases and it was a daring move to open the National Center for Newborn Screening and Management of Rare Diseases at the National Hospital of Pediatrics in 2013.

“At the time, Vu Tri Dung, Nguyen Ngoc Khanh, Can Bich Ngoc and Bui Phuong Thao were the first doctors who mentioned the concept of rare disease at the hospital and disseminated information in the community,” said Tran Mnh Dien, deputy director of the National Hospital of Pediatrics.

“In the last five years, the doctors at the center have been traveling all over the country giving lectures, providing knowledge about different types of rare diseases and discussing procedures for diagnosis, treatment and care for patients with these diseases.”

Fourteen years ago, no one had information about congenital metabolic disorders, and there was no therapy for it. However, the disease can be discovered through early screening and is curable 70 percent of the time.

“We make diagnoses, give advice on treatment, and ask for medical support, but also help patients and their family members to believe that they can defeat the disease,” Khanh said.

He said that some types of rare diseases are curable, while others need further research.

Fourteen years ago, no one had information about congenital metabolic disorders, and there was no therapy for it. However, the disease can be discovered through early screening and is curable 70 percent of the time. 

Patients who have Pompe, a disease related to Type II glycogen storage disorders, have to use enzymes to exist. In the past, the therapy was considered impossible in Vietnam because the enzyme was too expensive. Patients had to spend VND1-10 billion a year to buy the enzyme and use a drip the rest of their lives.

However, in the last four years, children can use the enzyme as doctors at the center are able to find drug donors from other countries.

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