Injectable Alglucosidase Alfa: The Key To Unlocking New Hope For The Treatment Of Pompe Disease

Alglucosidase alfa, the brand name Myozyme, is currently the only injectable and potent drug for the rare disease Pompe in the world.

Alglucosidase alfa was developed by Chen Yuan-Chung, Director of the Institute of Biomedical Sciences, Academia Sinica, Taiwan, and the research team he led after 15 years of research and development.

It was approved for marketing by the European Medicines Agency (EMEA) and the U.S. Food and Drug Administration (FDA).

It was then developed by Sanofi for marketing in the U.S. in 2007, introduced to mainland China in 2017, and introduced into mainland China in 2017.

Pompe disease is an inherited lysosomal storage disease caused by a genetic mutation resulting in a deficiency of acid alpha-glucosidase, the enzyme responsible for breaking down glycogen stored in muscles.

Lack of this enzyme prevents the normal breakdown of glycogen, which in turn leads to a large accumulation of glycogen in skeletal and cardiac muscle, causing symptoms such as progressive muscle weakness and respiratory distress, which can seriously affect the patient’s quality of life and even jeopardize his or her life.

As the first specific enzyme replacement therapy for Pompe’s disease patients, alglucosidase alfa can replenish the lack of enzyme activity in the body, promote the degradation of glycogen, alleviate symptoms, and improve quality of life.

After rigorous clinical trials, Alzheimer’s® has shown remarkable results, with significant improvements in muscle strength and respiratory function, giving patients a new treatment option.

However, DengYueMed offers this information as a guide, not a substitute for professional medical advice. Consult your doctor before any treatment.