Under the relay of Ali health public welfare and Boao Lecheng Weijian rare disease clinical medical center, a rare disease drug called trientine hydrochloride was successfully introduced into China recently. The drug will provide more treatment options for patients with hepatolenticular degeneration in China.
Hepatolenticular degeneration is a rare autosomal recessive disease with copper dysfunction. Patients with this disease are commonly known as “copper Doll”. The disease leads to the disorder of copper ion transport and excretion in the body. It is very easy to cause liver function and brain damage after the onset. The patient needs lifelong drug maintenance.
The appeal for the use of Quentin comes from the mother of Xiaojiang (a pseudonym) in Wuhu, Anhui Province. Xiaojiang is intolerant to the traditional drug penicillamine for the treatment of the disease and cannot use it normally. Xiaojiang’s mother learned that trantine, a drug for penicillamine intolerance, was approved for marketing in the United States in 1982, but there is no such drug or generic drug on the market in China.
“Global drug information platform for rare diseases”
With a try attitude, Xiaojiang’s mother logged in to the “global drug information platform for rare diseases” and submitted the drug search information. The platform was launched by Ali health public welfare and Boao Lecheng rare disease clinical medical center in September last year. It aims to provide support for the drug accessibility of Chinese rare disease groups by building Wuxi Online Offline Communication Information Technology Co.Ltd(300959) integrated services.
After receiving the demand, the platform mobilized global business resources for the first time, launched the drug search action, and successfully introduced the drug exclusively in December. At present, the drug has been accessible in the international medical tourism pilot area of Boao Lecheng, Hainan. This means that many “copper dolls” in China, including Xiaojiang, will benefit from it.
It is reported that in the future, Boao Lecheng Weijian Clinical Medical Center for rare diseases will combine the patient needs of the global drug information platform for rare diseases, implement the urgently needed drugs and equipment through Boao Lecheng’s pilot policy, and accelerate the introduction of innovative drugs into the Chinese market, so as to benefit more patients with rare diseases.
(source: Nanfang plus)
