< /mpsearch> Zhang Yan waited 26 months for this news. On April 6, 2022, Wang Jingjing, Deputy Chief Physician of the Department of Nephrology, Children’s Hospital Affiliated to Zhejiang University School of Medicine, told her that the first batch of drugs for the treatment of progeria in children had arrived at Hangzhou Xiaoshan Airport. After a week, everyone can go to the hospital to get medicine.” 
Photo source: provided by the family of the patient Kang put on his wings and hurried to Hangzhou. In 2014, Kangkang was diagnosed with childhood progeria (HGPS). There are few treatment options for the disease, and the world’s first (and so far only) specific drug, lonafarnib, was approved for marketing in the United States on November 20, 2020. From July 16, 2020, the Children’s Hospital Affiliated to Zhejiang University School of Medicine (hereinafter referred to as Zhejiang University Children’s Hospital) began to introduce drugs, and on April 25, 2022, the first Chinese child took lonafranil , which lasted 648 days. In the middle, there have been various twists and turns: when the hospital first submitted an application for clinical medication to the Provincial Health and Health Commission, it was rejected because they had not encountered any application for clinically urgently needed drugs by donation. I thought that after obtaining the approval of the State Food and Drug Administration, the drug could enter China smoothly, but I did not expect that overseas pharmaceutical companies have no experience in docking with the Chinese market, and Zhejiang University Children’s Hospital has to start all over the drug admission process; When the medicine is declared in China, if it is imported in the form of general trade, according to the customs requirements, the medicine needs to pay a 13% tax, which is 130,000 US dollars….In order to let Chinese children with progeria use as soon as possible In the past 26 months, the Children’s Hospital Affiliated to Zhejiang University School of Medicine, Zhejiang Provincial Drug Administration, Zhejiang Provincial Health Commission, Zhejiang Provincial Women’s Federation, Zhejiang Provincial Women’s and Children’s Foundation, Koide Rare Disease Center , Hong Kong Weijian Pharmaceutical Group and other parties have made great efforts. The health community has learned that Lonafranil will be given free of charge to Chinese children (before the drug is released in China) in the form of donated drugs, the first batch of drugs for 4 children for 4 months , the value is approximately equivalent to RMB 7.1199 million. This is the first time in mainland China, led by medical institutions, to solve the problem of urgently needed clinical medicines for children with rare diseases in my country through the temporary import route of urgently needed medicines. In the past, rare diseases in China lacked patients, doctors, money, and medicines. The “from 0 to 1” under the joint efforts of many parties may open up a new path for the availability of rare disease drugs.