U.S. company partners with Chinese firm to tackle rare disease
2017-09-27 10:18 Xinhua Editor: Gu Liping
Staff with U.S. company Amicus Therapeutics regularly travel to Shanghai to work out how to develop a new drug to treat Pompe disease, a rare muscle disorder.
John Crowley, chief executive of the company, led the recent trip to its Chinese partner WuXi Biologics, based in Shanghai.
Crowley said the new drug was expected to reduce the side effects of existing enzyme replacement therapy.
The disorder prevents the human body from making enough of an enzyme used by heart and muscle cells to convert a form of sugar called glycogen into energy. When glycogen builds up in these cells it can lead to swelling of the heart, disability and early death.
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