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Child patient with rare disease finds care in Lecheng

Updated: July 20, 2021 L M S

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Medical staff members prepare Cystagon for Xiao Li. 

The first prescription of Cystagon, a medicine used to treat cystinosis in children, was given to a 2-year-old patient at Hainan General Hospital's Lecheng branch in Hainan Boao Lecheng International Medical Tourism Pilot Zone on July 16.

The patient, named Xiao Li (alias), was diagnosed with cystinosis at around 1 year old after gene detection and the patient is currently the first of its kind from Chinese mainland to use Cystagon legally. 

"After taking the medicine for the first time in Lecheng, the patient is allowed to take a certain amount of the medicine home for further treatment according to the preferential policies in Lecheng," said Ji Xunqi, director of the pediatric department in the Lecheng branch of Hainan General Hospital. Ji Xunqi is also in charge of Xiao Li's case. 

"The licensed application of Cystagon in Lecheng will bring hope for other patients with cystinosis in the Chinese mainland and will also help medical staff learn about the latest treatment solutions and strengthen medical services in Hainan," added Ji.

Cystinosis is a rare, genetic, metabolic and lysosomal storage disease and its incidence rate is one out of 200,000 live births. It causes abnormal accumulation of the amino acid cystine in various organs and tissues in the body and causes extensive damage, especially to the eyes. 

Cystagon was approved by the US Food and Drug Administration to enter the market early on August 15, 1994. Clinical data has shown that the cysteine concentration in plasma can be effectively reduced within five to six hours after taking the medicine. 

Research has also shown that child patients taking the medicine with good renal function in the early stages of the disease will have a better chance of being cured.