[HIGHLIGHTS] The only drug is about to be withdrawn from China: rare disease patients encounter a rare moment

Xiaoxi was diagnosed with mucopolysaccharidosis IVA (MPS IVA) in July 2019. Zhang Yuqing's medicine is only enough until the end of July this year.

Traditional Chinese medicine conditioning, targeted treatment of complications through surgery, and even gene therapy are all within Zhang's plan. But these therapies have limited effect on her daughter's condition compared to specific drugs. Drug manufacturer BioMarin has decided not to renew the registration certificate for imported drugs in China, which will expire in May 2024.