In Depth: Fixing the High Cost of Rare Disease Treatment in China

By Liu Denghui, Wang Weijia and Cai Xuejiao

The heavy cost of rare disease treatment in China, such as this drug for spinal muscular atrophy, can be financially crippling to patients and their caregivers. Photo: VCG

Fourteen years ago, Tongtong was diagnosed with MPS II, a rare disease also known as Hunter syndrome that affects many different parts of the body. His condition gradually deteriorated due to a lack of specific treatments, and in late 2019, he became wheelchair-bound, and then he was even unable to stand.

Without an effective treatment, Tongtong’s condition is likely to continue to worsen and he could suffer from severe organ damage. His mother Liu Xia is desperate to find help.

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