Myofibrillar myopathy is a rare disease that causes muscle atrophy, stiffness, respiratory muscle weakness, and symptoms such as cardiomyopathy and movement disorders. Patients usually manifest symptoms of the disease during adolescence, but there is currently no effective treatment, and medical costs are extremely high.
A new drug developed by HKBU’s Centre for Chinese Herbal Medicine Drug Development has been designated as an “orphan drug” by the U.S. Food and Drug Administration (FDA) for the treatment of this rare disease, making it the first botanical drug in Hong Kong to receive such designation.
