Lele (pseudonym), a 3-year-old child suffering from homocystinuria, was successfully treated with the innovative medication Cystadane in the Boao Lecheng International Medical Tourism Pilot Zone (also known as Boao Hope City, one of the key industrial parks in China’s Hainan Province). This was the first time a patient in China has been treated with this medication.
Homocystinuria is an inherited disorder in which the body is unable to process certain building blocks of proteins (amino acids) properly. The most common form of homocystinuria is characterized by nearsightedness (myopia), dislocation of the lens at the front of the eye, an increased risk of abnormal blood clotting, and brittle bones that are prone to fracture (osteoporosis) or other skeletal abnormalities. As a methylating agent, betaine anhydrous, the active ingredient in Cystadane, can reduce the concentration of total homocysteine in the blood of patients with homocystinuria, thereby reducing symptoms and improving the psychomotor development ability of these patients.
After learning about Lele's situation, Lecheng applied to bring in the medication for use in the park, and received strong support from the Hainan Provincial Food and Drug Administration as well as the Lecheng Medical Supervision Bureau, quickly obtaining the required approval.
Thanks to a special policy allowing patients to take approved medications outside Hope City, after Lele took the innovative medicine for the first time in Lecheng, he was able to take the medicine at home where he was monitored according to his treatment needs, which greatly increased the convenience of his follow-up care.