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Made In China Gene Therapy A Father's Quest To Save Son With UltraRare Condition

A Journey Towards Gene Therapy For Ultra Rare Diseases In China

 
• By Brian Yang

Patient-driven drug development meets the old saying of "where there's a will, there's a way" in China, potentially the world's largest market for rare disease treatments.

Genetic engineering and gene manipulation concept. Hand is replacing part of a DNA molecule. 3D rendered illustration of DNA.
CAN CHINA DEVELOP ITS OWN GENE THERAPY? A PATIENT'S FATHER THINKS SO • Source: Shutterstock

When Jian Shu’s son Yue first started walking, he noticed that there was something not quite right, as the baby boy seemingly could not keep his body in balance.

Jian took Yue, whose name means happiness in Chinese, to see a doctor, who could not find what was wrong so ordered a gene sequencing test. The result showed that Yue had a type of progressive muscular dystrophy, a rare genetic condition that is usually inherited from a female carrier

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